SR One

Windward Bio is a clinical-stage biopharmaceutical company dedicated to enhancing outcomes for patients with advanced immunological diseases, focusing on severe respiratory conditions. The company is advancing a fully human monoclonal antibody targeting thymic stromal lymphopoietin (TSLP) into phase 2 clinical trials. This antibody, administered subcutaneously, potently binds to the TSLP ligand, extending its half-life and silencing its effector functions. Windward Bio is also developing innovative bispecific programs for other immunological disorders, led by a team of experienced biopharmaceutical executives aiming to address significant unmet medical needs in this field.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Avenzo Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative therapies for oncology, specifically targeting underserved and rare cancers. The company operates a specialized platform that focuses on creating novel treatments aimed at genomically defined cancer types. By prioritizing targeted therapies, Avenzo Therapeutics seeks to advance cancer treatment options for patients who may not have access to effective solutions, thus addressing significant gaps in the current therapeutic landscape.

Nomic is a bioengineering company focused on enhancing access to the human proteome, similar to the accessibility of the human genome. The company is developing the next-generation ELISA platform, known as nELISA, which is designed for high-throughput protein measurement. Nomic's solutions enable scientists to conduct protein profiling efficiently, allowing them to determine the scope and scale of their experiments. The platform features a cost-effective reagent system that integrates seamlessly with existing industry cytometers, facilitating high-throughput analysis and the creation of custom protein panels. By leveraging advanced technology, Nomic aims to empower researchers and nanotechnology developers to utilize DNA as a flexible and programmable linker, effectively addressing challenges such as cross-reactivity in protein measurements.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for eye diseases using its proprietary Mucus Penetrating Particles (MPP) technology. This technology enhances drug distribution and pharmacokinetics by coating mucosal surfaces with biocompatible, drug-loaded particles, allowing for improved local drug concentrations while minimizing systemic exposure. Kala's lead product candidate, KPI-121 0.25%, has completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS has also completed two Phase III trials for managing inflammation and pain after ocular surgery. The company is advancing KPI-285, a receptor tyrosine kinase inhibitor, in preclinical studies targeting retinal diseases. Established in 2009 and headquartered in Watertown, Massachusetts, Kala Pharmaceuticals continues to explore diverse product opportunities while focusing on internal development and research collaborations to address significant clinical needs.

Zenas BioPharma is a clinical-stage biopharmaceutical company headquartered in Florida, USA, that specializes in developing immune-based therapies for patients. The company’s lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to target both CD19 and FcγRIIb, which are found in B cell lineages. This innovative approach aims to inhibit the activity of cells involved in various autoimmune diseases while preserving their presence, thus avoiding depletion. Through its focus on these advanced therapies, Zenas BioPharma seeks to address unmet medical needs in the treatment of autoimmune conditions.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

Alumis is a clinical-stage biopharmaceutical company dedicated to developing precision medicines for autoimmune disorders. By leveraging a proprietary data analytics platform and biological insights, Alumis aims to transform the treatment landscape for patients suffering from these conditions. The company focuses on identifying and advancing therapeutics based on genetically validated targets, which enables the creation of targeted therapies that significantly improve patient outcomes. With a team of experts experienced in drug discovery and immunology, Alumis seeks to replace broad immunosuppression with more effective, targeted treatment options, ultimately enhancing the quality of life for individuals affected by autoimmune diseases.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

Avalyn Pharma, Inc. is a biopharmaceutical company focused on developing innovative therapies for idiopathic pulmonary fibrosis (IPF) and other serious respiratory conditions. Headquartered in Seattle, Washington, with an additional office in San Diego, the company specializes in an inhaled formulation of pirfenidone, known as AP01. This formulation aims to enhance the amount of medication that reaches the lungs while minimizing side effects associated with oral treatments. Established in 2011, Avalyn Pharma is dedicated to improving care and outcomes for patients with severe respiratory diseases through its advanced therapeutic pipeline and novel inhaled therapeutics. Initially named Genoa Pharmaceuticals, the company rebranded to its current name in July 2017.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

Avenzo Therapeutics is a clinical-stage biotechnology company dedicated to developing innovative therapies for oncology, specifically targeting underserved and rare cancers. The company operates a specialized platform that focuses on creating novel treatments aimed at genomically defined cancer types. By prioritizing targeted therapies, Avenzo Therapeutics seeks to advance cancer treatment options for patients who may not have access to effective solutions, thus addressing significant gaps in the current therapeutic landscape.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

Pulmocide Ltd is a biotechnology company based in London, United Kingdom, that specializes in the discovery and development of inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. Founded in 2007, Pulmocide focuses on creating a new generation of antifungal drugs specifically designed for inhaled administration. This method maximizes the delivery of medication directly to the lungs while reducing systemic exposure, thereby minimizing potential toxicities. The company's innovative treatments are intended to offer superior efficacy against respiratory aspergillosis and provide safer, more effective options for healthcare providers managing acute and chronic respiratory diseases.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Ancora Biotech is a biotechnology company dedicated to the development of antibody biotherapeutics aimed at treating several life-threatening diseases. The company specializes in providing clinical development support for therapies targeting B cell cancers, as well as inflammatory and metabolic disorders, and chronic hepatitis B infections. By focusing on these critical areas, Ancora Biotech seeks to enable healthcare professionals to effectively treat patients suffering from serious health conditions.

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

Nomic is a bioengineering company focused on enhancing access to the human proteome, similar to the accessibility of the human genome. The company is developing the next-generation ELISA platform, known as nELISA, which is designed for high-throughput protein measurement. Nomic's solutions enable scientists to conduct protein profiling efficiently, allowing them to determine the scope and scale of their experiments. The platform features a cost-effective reagent system that integrates seamlessly with existing industry cytometers, facilitating high-throughput analysis and the creation of custom protein panels. By leveraging advanced technology, Nomic aims to empower researchers and nanotechnology developers to utilize DNA as a flexible and programmable linker, effectively addressing challenges such as cross-reactivity in protein measurements.

Odyssey Therapeutics is a biotechnology company focused on the development of next-generation immunomodulators and oncology medicines. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, unlocking high-potential targets while reducing the risk of attrition during the development process. Odyssey is committed to advancing precision medicine in the healthcare sector, with a product pipeline designed to facilitate a faster transition to clinical development, thereby improving treatment options for patients with various health conditions.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Established in 2019, the company specializes in genetic medicine by utilizing a proprietary platform that employs endogenous enzymes, specifically adenosine deaminase acting on RNA (ADAR), to precisely target and correct single point mutations in RNA transcripts. This innovative approach aims to restore the production of functional proteins, offering potential treatments for a variety of diseases, including those affecting genetic, cardiometabolic, complement-mediated, and central nervous system areas. ADARx Pharmaceuticals is dedicated to developing life-saving therapeutics that address genetic mutations and provide solutions for conditions that are currently considered incurable.

eFFECTOR Therapeutics, Inc. is a clinical-stage biotechnology company based in San Diego, California, that specializes in the development of selective translation regulators for the treatment of cancer and other serious diseases. Founded in 2012, the company is pioneering a new class of oncology drugs known as selective translation regulator inhibitors (STRIs). These drug candidates specifically target the eIF4F complex and its associated kinase, MNK 1/2, which play critical roles in protein synthesis and are linked to prominent cancer signaling pathways such as PI3K-AKT and RAS-MEK. eFFECTOR's products aim to inhibit pathways that drive tumor growth, survival, and immune evasion by regulating the expression of oncoproteins and immunosuppressive proteins. The lead candidate, tomivosertib, is currently being evaluated in a Phase 2b trial for non-small cell lung cancer in combination with pembrolizumab. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation in a Phase 1/2 trial, with further expansion cohorts anticipated. The company also collaborates with Pfizer to advance inhibitors targeting eIF4E.

Pulmocide Ltd is a biotechnology company based in London, United Kingdom, that specializes in the discovery and development of inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. Founded in 2007, Pulmocide focuses on creating a new generation of antifungal drugs specifically designed for inhaled administration. This method maximizes the delivery of medication directly to the lungs while reducing systemic exposure, thereby minimizing potential toxicities. The company's innovative treatments are intended to offer superior efficacy against respiratory aspergillosis and provide safer, more effective options for healthcare providers managing acute and chronic respiratory diseases.

BioTheryX, Inc. is a clinical-stage biopharmaceutical company based in Chappaqua, New York, that specializes in restoring protein homeostasis through innovative small molecule therapies. The company develops Protein Homeostatic Modulators, including a small molecular glue that targets cereblon for protein degradation, with applications in oncology, inflammation, and other diseases. BioTheryX employs its proprietary PRODEGY platform, which utilizes a library of cereblon binders to design targeted protein degraders (TPDs). Among its key products is BTX-A51, an oral multi-kinase inhibitor aimed at treating specific leukemic stem cell targets and preventing the transcription of oncogenic genes. Founded in 2007, BioTheryX aims to address unmet medical needs by leveraging its expertise in targeted protein degradation.

Arcellx is a clinical-stage biotechnology company focused on developing innovative immunotherapies, particularly in the realm of cell therapy, for patients with cancer and other serious diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Arcellx's lead product candidate, CART-ddBCMA, is currently being investigated in a Phase 1 clinical trial for the treatment of relapsed or refractory multiple myeloma. This therapy has received several designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is progressing its dosable and controllable CAR-T therapy, known as ARC-SparX, through two clinical programs targeting relapsed or refractory multiple myeloma and acute myeloid leukemia. Through these efforts, Arcellx seeks to contribute significantly to the advancement of cell therapy in modern medicine.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

F-Star Therapeutics is a biopharmaceutical company focused on developing novel bispecific antibody products aimed at improving upon current standards of care, particularly in cancer therapy. The company is uniquely positioned with a strong patent portfolio, enabling it to create and develop Fcab antibody fragments and bispecific antibodies by modifying the constant region of an antibody. F-Star's Modular Antibody Technology allows for rapid discovery and development of bispecific antibodies by adding binding sites to the constant region, facilitating easier production compared to other antibody formats. The company is leveraging this technology to build a pipeline of product candidates. Founded in 2006, F-Star has received significant funding from leading venture capital firms and strategic corporate investors. The company operates out of Cambridge, UK, with over 35 employees dedicated to research and development.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments for cancer patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of targeted therapies that address unmet medical needs in oncology. Their drug development process involves replicating naturally occurring mutations in laboratory settings, allowing for the testing of these mutations' effects on signaling pathways and their responsiveness to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharmaceutical companies expand the subgroups of cancer patients who may benefit from existing therapies.

Palleon Pharmaceuticals Inc. is a biotechnology company dedicated to developing innovative therapeutics that target glycan-mediated immune regulation for the treatment of cancer and inflammatory diseases. Founded in 2015 and based in Waltham, Massachusetts, the company specializes in glycoimmune checkpoint inhibitors, leveraging advanced insights from glycoscience and human immunology. Palleon's biotechnology platform aims to provide a novel approach to cancer treatment by targeting various immune cell types, thus offering physicians an expanded array of combination therapies to enhance treatment efficacy and address resistance to existing immuno-oncology agents. In addition to cancer, the company also focuses on therapeutics for inflammatory diseases, including autoimmunity and fibrosis.

Pandion Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, focused on developing innovative therapeutics for patients with autoimmune diseases. The company’s lead product candidate, PT101, is an engineered interleukin-2 variant currently undergoing Phase 1a clinical trials for various autoimmune and inflammatory conditions, including moderate-to-severe ulcerative colitis and systemic lupus erythematosus. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to provide tissue-selective immunomodulation by targeting specific adhesion molecules in the gastrointestinal tract. Pandion employs a network-based approach to immune system conceptualization through its TALON drug design platform and collaborates with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. The company aims to create a new generation of therapeutics that offer enhanced efficacy and safety for the treatment of autoimmune and inflammatory diseases.

Design Therapeutics, Inc. is a clinical-stage biotechnology company headquartered in Solana Beach, California, focused on developing innovative therapies for serious degenerative disorders caused by nucleotide repeat expansions. The company specializes in a novel class of small-molecule gene-targeted chimera therapeutics known as GeneTACTM molecules, which aim to modify disease progression by addressing the root causes of these conditions. Its lead program targets Friedreich’s ataxia, while additional efforts are directed toward conditions such as Fuchs endothelial corneal dystrophy and other degenerative diseases like Fragile X syndrome and myotonic dystrophy. Since its incorporation in 2017, Design Therapeutics has been dedicated to advancing its therapeutic candidates to potentially transform treatment options for patients suffering from these inherited disorders.

Arcellx is a clinical-stage biotechnology company focused on developing innovative immunotherapies, particularly in the realm of cell therapy, for patients with cancer and other serious diseases. The company aims to enhance the safety, effectiveness, and accessibility of these therapies. Arcellx's lead product candidate, CART-ddBCMA, is currently being investigated in a Phase 1 clinical trial for the treatment of relapsed or refractory multiple myeloma. This therapy has received several designations from the U.S. Food and Drug Administration, including Fast Track and Orphan Drug status. Additionally, Arcellx is progressing its dosable and controllable CAR-T therapy, known as ARC-SparX, through two clinical programs targeting relapsed or refractory multiple myeloma and acute myeloid leukemia. Through these efforts, Arcellx seeks to contribute significantly to the advancement of cell therapy in modern medicine.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

Turning Point Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on designing and developing novel small molecule targeted therapies for oncology. The company specializes in creating next-generation tyrosine kinase inhibitors (TKIs) that address genetic drivers of cancer, catering to both TKI-naïve and TKI-pretreated patients. Its lead drug candidate, repotrectinib, is currently undergoing evaluation in the Phase I/II TRIDENT-1 trial for the treatment of patients with ROS1+ advanced non-small-cell lung cancer and those with ROS1+, NTRK+, or ALK+ advanced solid tumors. Additionally, Turning Point Therapeutics is advancing a pipeline that includes other multi-targeted drug candidates, such as TPX-0046, a RET/SRC inhibitor; TPX-0022, a MET/CSF1R/SRC inhibitor; and TPX-0131, a preclinical ALK inhibitor. Founded in 2013, the company is dedicated to addressing the limitations of existing therapies through precision medicine in oncology.

Gotham Therapeutics Corp. is a biotechnology company based in New York, founded in 2017, that focuses on developing innovative treatments for patients with cancers, autoimmune disorders, and neurodegenerative diseases. The company specializes in a novel class of drugs that target the machinery of epitranscriptomics, which involves altering the activity of proteins that modify messenger ribonucleic acid (mRNA). By utilizing small molecules that inhibit the transcription of proteins through modifications to mRNA, Gotham Therapeutics aims to enhance treatment options and improve outcomes for individuals affected by serious health conditions.

Morphic Therapeutic is a biotechnology company focused on developing oral integrin therapies to address serious chronic diseases such as autoimmune disorders, cardiovascular issues, metabolic diseases, fibrosis, and cancer. The company leverages its expertise in integrins to create a pipeline of small-molecule inhibitors targeting various integrin pathways. This innovative approach aims to provide effective treatments for patients suffering from immunological, fibrotic, neoplastic, and vascular conditions, contributing to advancements in biopharmaceuticals.

Bicycle Therapeutics plc is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, specializing in the development of a novel class of medicines known as Bicycles. These are synthetic short peptides designed to form two loops that stabilize their structure. The company's lead product candidate, BT1718, is a Bicycle Toxin Conjugate (BTC) currently undergoing Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also a BTC in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies targeting Nectin-4. Additionally, Bicycle is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to address therapeutic areas such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. Bicycle Therapeutics was incorporated in 2009 and aims to address significant unmet medical needs in oncology and beyond.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.

Zikani Therapeutics, Inc. is a biotechnology company focused on developing and commercializing therapeutics for patients with limited treatment options. The company utilizes its TURBO-ZM platform to create novel ribosome modulating agents (RMAs) aimed at treating a range of conditions, including cystic fibrosis, familial adenomatous polyposis, APC mutant colon cancer, recessive dystrophic epidermolysis bullosa, rare cancers, and other rare genetic diseases. Founded in 2014 and originally known as Macrolide Pharmaceuticals, Zikani is headquartered in Watertown, Massachusetts. The company's antibiotic compounds leverage advancements in synthetic chemistry and microbiology to address serious, multi-drug resistant gram-negative infections, which are a significant cause of severe health issues in the United States. Through its innovative approach, Zikani Therapeutics seeks to enhance patient outcomes and expand therapeutic options for challenging medical conditions.

Pandion Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, focused on developing innovative therapeutics for patients with autoimmune diseases. The company’s lead product candidate, PT101, is an engineered interleukin-2 variant currently undergoing Phase 1a clinical trials for various autoimmune and inflammatory conditions, including moderate-to-severe ulcerative colitis and systemic lupus erythematosus. In addition to PT101, Pandion's pipeline includes PT627, a systemic PD-1 agonist in preclinical studies, and two bifunctional molecules, PT001 and PT002, designed to provide tissue-selective immunomodulation by targeting specific adhesion molecules in the gastrointestinal tract. Pandion employs a network-based approach to immune system conceptualization through its TALON drug design platform and collaborates with Astellas Pharma to develop locally acting immunomodulators for pancreatic autoimmune diseases. The company aims to create a new generation of therapeutics that offer enhanced efficacy and safety for the treatment of autoimmune and inflammatory diseases.

Atox Bio Inc. is a biotechnology company based in Ness Ziona, Israel, with an additional office in North Carolina. Founded in 2003, the company specializes in developing immunomodulators aimed at treating critically ill patients facing severe acute inflammation due to infections. Atox Bio focuses on novel modulators of the immune response, targeting both adaptive and innate pathways that play crucial roles in managing infectious and inflammatory diseases, as well as ischemia/reperfusion injuries. The company's products are designed to improve outcomes for patients suffering from rare, life-threatening infections, demonstrating significant improvements in various clinical endpoints. By enhancing the host's ability to combat infections and minimizing tissue damage during acute inflammatory events, Atox Bio aims to address serious unmet medical needs in critical care settings.

Palleon Pharmaceuticals Inc. is a biotechnology company dedicated to developing innovative therapeutics that target glycan-mediated immune regulation for the treatment of cancer and inflammatory diseases. Founded in 2015 and based in Waltham, Massachusetts, the company specializes in glycoimmune checkpoint inhibitors, leveraging advanced insights from glycoscience and human immunology. Palleon's biotechnology platform aims to provide a novel approach to cancer treatment by targeting various immune cell types, thus offering physicians an expanded array of combination therapies to enhance treatment efficacy and address resistance to existing immuno-oncology agents. In addition to cancer, the company also focuses on therapeutics for inflammatory diseases, including autoimmunity and fibrosis.

eFFECTOR Therapeutics, Inc. is a clinical-stage biotechnology company based in San Diego, California, that specializes in the development of selective translation regulators for the treatment of cancer and other serious diseases. Founded in 2012, the company is pioneering a new class of oncology drugs known as selective translation regulator inhibitors (STRIs). These drug candidates specifically target the eIF4F complex and its associated kinase, MNK 1/2, which play critical roles in protein synthesis and are linked to prominent cancer signaling pathways such as PI3K-AKT and RAS-MEK. eFFECTOR's products aim to inhibit pathways that drive tumor growth, survival, and immune evasion by regulating the expression of oncoproteins and immunosuppressive proteins. The lead candidate, tomivosertib, is currently being evaluated in a Phase 2b trial for non-small cell lung cancer in combination with pembrolizumab. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation in a Phase 1/2 trial, with further expansion cohorts anticipated. The company also collaborates with Pfizer to advance inhibitors targeting eIF4E.

Turning Point Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on designing and developing novel small molecule targeted therapies for oncology. The company specializes in creating next-generation tyrosine kinase inhibitors (TKIs) that address genetic drivers of cancer, catering to both TKI-naïve and TKI-pretreated patients. Its lead drug candidate, repotrectinib, is currently undergoing evaluation in the Phase I/II TRIDENT-1 trial for the treatment of patients with ROS1+ advanced non-small-cell lung cancer and those with ROS1+, NTRK+, or ALK+ advanced solid tumors. Additionally, Turning Point Therapeutics is advancing a pipeline that includes other multi-targeted drug candidates, such as TPX-0046, a RET/SRC inhibitor; TPX-0022, a MET/CSF1R/SRC inhibitor; and TPX-0131, a preclinical ALK inhibitor. Founded in 2013, the company is dedicated to addressing the limitations of existing therapies through precision medicine in oncology.

Pulmocide Ltd is a biotechnology company based in London, United Kingdom, that specializes in the discovery and development of inhaled medicines aimed at treating serious viral and fungal infections of the respiratory tract. Founded in 2007, Pulmocide focuses on creating a new generation of antifungal drugs specifically designed for inhaled administration. This method maximizes the delivery of medication directly to the lungs while reducing systemic exposure, thereby minimizing potential toxicities. The company's innovative treatments are intended to offer superior efficacy against respiratory aspergillosis and provide safer, more effective options for healthcare providers managing acute and chronic respiratory diseases.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

True North Therapeutics is a biotechnology company based in South San Francisco, California, founded in 2013 as a spin-out of iPierian. The company focuses on developing therapies that target the classical pathway of the immune system's complement system to address rare diseases with high unmet clinical needs. Its lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway, aiming to treat conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, antibody-mediated rejection, bullous pemphigoid, and other rare diseases in hematologic, kidney transplant, dermatology, and neurological spaces. True North Therapeutics was acquired by Bioverativ Inc.

Bicycle Therapeutics plc is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, specializing in the development of a novel class of medicines known as Bicycles. These are synthetic short peptides designed to form two loops that stabilize their structure. The company's lead product candidate, BT1718, is a Bicycle Toxin Conjugate (BTC) currently undergoing Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also a BTC in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies targeting Nectin-4. Additionally, Bicycle is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to address therapeutic areas such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. Bicycle Therapeutics was incorporated in 2009 and aims to address significant unmet medical needs in oncology and beyond.

Arch Oncology, Inc. is an immuno-oncology company focused on discovering and developing antibody therapeutics for the treatment of solid tumors and hematologic cancers. Founded in 2006 and headquartered in Brisbane, California, with a scientific research laboratory in St. Louis, Missouri, the company is known for its innovative approach to cancer treatment. Arch Oncology specializes in anti-CD47 antibodies, which represent a new class of checkpoint inhibitors that engage both the adaptive and innate immune responses. These functionally diverse antibodies not only inhibit immune checkpoints but also selectively target and kill tumor cells, providing patients with new therapeutic options in the fight against cancer.

Morphic Therapeutic is a biotechnology company focused on developing oral integrin therapies to address serious chronic diseases such as autoimmune disorders, cardiovascular issues, metabolic diseases, fibrosis, and cancer. The company leverages its expertise in integrins to create a pipeline of small-molecule inhibitors targeting various integrin pathways. This innovative approach aims to provide effective treatments for patients suffering from immunological, fibrotic, neoplastic, and vascular conditions, contributing to advancements in biopharmaceuticals.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

eFFECTOR Therapeutics, Inc. is a clinical-stage biotechnology company based in San Diego, California, that specializes in the development of selective translation regulators for the treatment of cancer and other serious diseases. Founded in 2012, the company is pioneering a new class of oncology drugs known as selective translation regulator inhibitors (STRIs). These drug candidates specifically target the eIF4F complex and its associated kinase, MNK 1/2, which play critical roles in protein synthesis and are linked to prominent cancer signaling pathways such as PI3K-AKT and RAS-MEK. eFFECTOR's products aim to inhibit pathways that drive tumor growth, survival, and immune evasion by regulating the expression of oncoproteins and immunosuppressive proteins. The lead candidate, tomivosertib, is currently being evaluated in a Phase 2b trial for non-small cell lung cancer in combination with pembrolizumab. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation in a Phase 1/2 trial, with further expansion cohorts anticipated. The company also collaborates with Pfizer to advance inhibitors targeting eIF4E.

Navitor Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing small molecule drugs that target specific nutrient-sensing proteins to modulate mTORC1 activity. This master regulator of cell growth and metabolism plays a crucial role in various age-related diseases. The company's proprietary platform allows for both selective inhibition and activation of mTORC1, offering potential treatments for conditions such as chronic kidney disease, depression, and other metabolic, neurodegenerative, autoimmune, and musculoskeletal diseases. Notable compounds include NV-5138, which activates mTORC1 and modulates sestrin, a natural activator of mTORC1, and the NValog program, which targets chronic kidney disease through selective mTORC1 inhibition. Founded in 2009, Navitor Pharmaceuticals aims to unlock the therapeutic potential of the mTOR pathway for a wide range of diseases.

True North Therapeutics is a biotechnology company based in South San Francisco, California, founded in 2013 as a spin-out of iPierian. The company focuses on developing therapies that target the classical pathway of the immune system's complement system to address rare diseases with high unmet clinical needs. Its lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway, aiming to treat conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, antibody-mediated rejection, bullous pemphigoid, and other rare diseases in hematologic, kidney transplant, dermatology, and neurological spaces. True North Therapeutics was acquired by Bioverativ Inc.

Puridify Limited is a bioprocessing company based in Stevenage, United Kingdom, founded in 2013 by Tom Haywood, Oliver Hardick, and Iwan Roberts. The company specializes in developing advanced purification technologies for the manufacturing of industrial biomolecules. Its flagship product, FibroSelect, utilizes a unique nanofiber-based platform that enhances purification efficiency while addressing the limitations found in traditional packed bed chromatography systems. This innovative technology is designed to provide consistent purification performance across various scales, making it suitable for the purification of complex biotherapeutics, including gene therapies, viral vectors, and fusion proteins. By improving the efficiency and cost-effectiveness of biopharmaceutical production, Puridify aims to advance the industry and support the growing demand for high-quality biomolecule products.

Decibel Therapeutics, Inc. is a clinical-stage biotechnology company focused on discovering and developing innovative therapies aimed at protecting, repairing, and restoring hearing. The company addresses various hearing disorders, including those related to pediatric cancers, cystic fibrosis, noise exposure, age-related and genetic hearing loss, as well as conditions characterized by persistent tinnitus. Decibel Therapeutics employs a multifaceted approach that includes drug delivery to the inner ear, advanced imaging techniques, and pharmacokinetic and pharmacodynamic modeling. The company leverages its expertise in inner ear biology, single-cell genomics, and bioinformatics to develop effective treatments for hearing loss and related disorders. Established in 2013 and headquartered in Boston, Massachusetts, Decibel Therapeutics was originally known as Hearing, Inc. before rebranding in 2014.

Principia Biopharma Inc. is a late-stage biopharmaceutical company headquartered in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead product, rilzabrutinib, is an inhibitor undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 trials for immune thrombocytopenia and Phase 2 trials for IgG4-related disease. Additionally, Principia is developing PRN2246/SAR442168, which is in Phase II for multiple sclerosis and other central nervous system conditions, alongside PRN473, a drug candidate in Phase I trials for immune-mediated diseases. Principia Biopharma maintains a collaboration agreement with Genzyme Corporation for the development of treatments for MS and other CNS diseases. Originally incorporated in 2008, the company is dedicated to addressing significant unmet medical needs in immunology and oncology through its innovative drug development platform.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Akamis Bio is a clinical-stage oncology company dedicated to advancing cancer treatment through its innovative Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform. The company develops a portfolio of therapeutics specifically targeting solid tumors, aiming to enhance patients' immune responses to recognize and eradicate cancer cells. By harnessing a proprietary intravenously administered viral platform, Akamis Bio focuses on delivering effective gene therapies that address the needs of cancer patients, striving to improve their quality of life and treatment outcomes.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

CRISPR Therapeutics AG is a biotechnology company based in Zug, Switzerland, dedicated to developing transformative gene-based medicines for serious human diseases using CRISPR/Cas9 technology. This revolutionary gene-editing platform allows for precise changes to genomic DNA. The company's portfolio includes therapeutic programs targeting hemoglobinopathies, oncology, regenerative medicine, and rare diseases. Their lead product candidate is CTX001, a gene-edited therapy for transfusion-dependent beta thalassemia or severe sickle cell disease, which has been approved as Casgevy in collaboration with Vertex Pharmaceuticals. Additionally, CRISPR Therapeutics is developing CTX110, an allogeneic CAR-T therapy targeting CD19-positive malignancies, and other programs like CTX120 for multiple myeloma and CTX130 for solid tumors and hematologic malignancies. The company also engages in regenerative medicine programs for diabetes and genetic disease treatments such as glycogen storage disease Ia, Duchenne muscular dystrophy, and cystic fibrosis. CRISPR Therapeutics has strategic partnerships with Bayer Healthcare LLC, Vertex Pharmaceuticals Incorporated, and ViaCyte, Inc.

True North Therapeutics is a biotechnology company based in South San Francisco, California, founded in 2013 as a spin-out of iPierian. The company focuses on developing therapies that target the classical pathway of the immune system's complement system to address rare diseases with high unmet clinical needs. Its lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway, aiming to treat conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, antibody-mediated rejection, bullous pemphigoid, and other rare diseases in hematologic, kidney transplant, dermatology, and neurological spaces. True North Therapeutics was acquired by Bioverativ Inc.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Zikani Therapeutics, Inc. is a biotechnology company focused on developing and commercializing therapeutics for patients with limited treatment options. The company utilizes its TURBO-ZM platform to create novel ribosome modulating agents (RMAs) aimed at treating a range of conditions, including cystic fibrosis, familial adenomatous polyposis, APC mutant colon cancer, recessive dystrophic epidermolysis bullosa, rare cancers, and other rare genetic diseases. Founded in 2014 and originally known as Macrolide Pharmaceuticals, Zikani is headquartered in Watertown, Massachusetts. The company's antibiotic compounds leverage advancements in synthetic chemistry and microbiology to address serious, multi-drug resistant gram-negative infections, which are a significant cause of severe health issues in the United States. Through its innovative approach, Zikani Therapeutics seeks to enhance patient outcomes and expand therapeutic options for challenging medical conditions.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for treating various cancers and autoimmune diseases. Unlike T-cell therapies, NK cells have an inherent ability to recognize and destroy abnormal cells without genetic alteration, offering promise for diverse tumor types with potentially better tolerated side effects. Nkarta's approach involves leveraging its NK expansion platform technology alongside proprietary cell engineering techniques to generate a substantial supply of enhanced NK cells that can persistently target and eliminate cancer cells.

Avhana Health Inc., established in 2014 and based in Baltimore, Maryland, specializes in streamlining healthcare provider workflows through its application. This platform integrates with electronic health records (EHR) to deliver real-time, patient-specific clinical recommendations, actionable guidelines, and performance metrics directly within the EHR interface. The software facilitates pre-visit planning, suggests relevant orders that can be submitted directly into the EHR, and tracks provider activity for performance evaluation. Avhana Health's mission is to empower healthcare teams with impactful, patient-specific support to enhance care quality and reduce costs.

Aileron Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, dedicated to developing innovative therapeutics, particularly stabilized cell-permeating alpha-helical peptides, for oncology and other therapeutic areas. The company’s lead product candidate, ALRN-6924, is designed to treat advanced solid tumors and is currently undergoing various clinical trials, including Phase 2a studies for peripheral T-cell lymphoma and advanced solid tumors, as well as Phase 1 trials for acute myeloid leukemia and myelodysplastic syndromes. Aileron Therapeutics also explores next-generation wild type p53 reactivators and has established collaborations with notable institutions such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer to evaluate the effectiveness of ALRN-6924 in combination with other treatments. Founded in 2001 and originally known as Renegade Therapeutics, the company aims to address critical cell functions through its novel approach to modulating protein-protein interactions.

Bicycle Therapeutics plc is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, specializing in the development of a novel class of medicines known as Bicycles. These are synthetic short peptides designed to form two loops that stabilize their structure. The company's lead product candidate, BT1718, is a Bicycle Toxin Conjugate (BTC) currently undergoing Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also a BTC in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies targeting Nectin-4. Additionally, Bicycle is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to address therapeutic areas such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. Bicycle Therapeutics was incorporated in 2009 and aims to address significant unmet medical needs in oncology and beyond.

Atopix Therapeutics Limited is a clinical-stage biopharmaceutical company based in Oxford, United Kingdom, focused on developing innovative anti-inflammatory medicines for the treatment of asthma, chronic allergic conditions, and other atopic diseases. Founded in 2012, the company specializes in a novel class of oral anti-allergic medications known as CRTH2 antagonists. Its lead candidate is currently undergoing a Phase 2 clinical trial aimed at addressing moderate to severe atopic dermatitis, with studies being conducted at several prominent dermatology centers across Europe. Atopix Therapeutics operates as a subsidiary of Chiesi Farmaceutici S.p.A.

Atox Bio Inc. is a biotechnology company based in Ness Ziona, Israel, with an additional office in North Carolina. Founded in 2003, the company specializes in developing immunomodulators aimed at treating critically ill patients facing severe acute inflammation due to infections. Atox Bio focuses on novel modulators of the immune response, targeting both adaptive and innate pathways that play crucial roles in managing infectious and inflammatory diseases, as well as ischemia/reperfusion injuries. The company's products are designed to improve outcomes for patients suffering from rare, life-threatening infections, demonstrating significant improvements in various clinical endpoints. By enhancing the host's ability to combat infections and minimizing tissue damage during acute inflammatory events, Atox Bio aims to address serious unmet medical needs in critical care settings.

Navitor Pharmaceuticals is a biopharmaceutical company based in Cambridge, Massachusetts, focused on developing small molecule drugs that target specific nutrient-sensing proteins to modulate mTORC1 activity. This master regulator of cell growth and metabolism plays a crucial role in various age-related diseases. The company's proprietary platform allows for both selective inhibition and activation of mTORC1, offering potential treatments for conditions such as chronic kidney disease, depression, and other metabolic, neurodegenerative, autoimmune, and musculoskeletal diseases. Notable compounds include NV-5138, which activates mTORC1 and modulates sestrin, a natural activator of mTORC1, and the NValog program, which targets chronic kidney disease through selective mTORC1 inhibition. Founded in 2009, Navitor Pharmaceuticals aims to unlock the therapeutic potential of the mTOR pathway for a wide range of diseases.

True North Therapeutics is a biotechnology company based in South San Francisco, California, founded in 2013 as a spin-out of iPierian. The company focuses on developing therapies that target the classical pathway of the immune system's complement system to address rare diseases with high unmet clinical needs. Its lead product, TNT009, is a monoclonal antibody designed to selectively inhibit the classical complement pathway, aiming to treat conditions such as cold agglutinin disease, warm autoimmune hemolytic anemia, antibody-mediated rejection, bullous pemphigoid, and other rare diseases in hematologic, kidney transplant, dermatology, and neurological spaces. True North Therapeutics was acquired by Bioverativ Inc.

Puridify Limited is a bioprocessing company based in Stevenage, United Kingdom, founded in 2013 by Tom Haywood, Oliver Hardick, and Iwan Roberts. The company specializes in developing advanced purification technologies for the manufacturing of industrial biomolecules. Its flagship product, FibroSelect, utilizes a unique nanofiber-based platform that enhances purification efficiency while addressing the limitations found in traditional packed bed chromatography systems. This innovative technology is designed to provide consistent purification performance across various scales, making it suitable for the purification of complex biotherapeutics, including gene therapies, viral vectors, and fusion proteins. By improving the efficiency and cost-effectiveness of biopharmaceutical production, Puridify aims to advance the industry and support the growing demand for high-quality biomolecule products.

Principia Biopharma Inc. is a late-stage biopharmaceutical company headquartered in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead product, rilzabrutinib, is an inhibitor undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 trials for immune thrombocytopenia and Phase 2 trials for IgG4-related disease. Additionally, Principia is developing PRN2246/SAR442168, which is in Phase II for multiple sclerosis and other central nervous system conditions, alongside PRN473, a drug candidate in Phase I trials for immune-mediated diseases. Principia Biopharma maintains a collaboration agreement with Genzyme Corporation for the development of treatments for MS and other CNS diseases. Originally incorporated in 2008, the company is dedicated to addressing significant unmet medical needs in immunology and oncology through its innovative drug development platform.

Spero Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative treatments for multi-drug resistant (MDR) bacterial infections and rare diseases. Founded in 2013, the company is advancing several product candidates, including tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic aimed at treating MDR gram-negative infections in adults. Additionally, Spero is developing SPR206, an intravenous agent for MDR gram-negative infections in hospital settings, and SPR720, an oral antibiotic targeting pulmonary non-tuberculous mycobacterial disease. The company collaborates with various partners, including Meiji Seika Pharma to support tebipenem HBr's development and the Bill & Melinda Gates Medical Research Institute for SPR720. Spero Therapeutics employs a focused approach to drug development, emphasizing novel mechanisms to address the urgent need for effective therapeutics against serious bacterial infections.

Alios BioPharma is focused on discovering and developing innovative antiviral therapies aimed at treating both acute and chronic viral infections. The company utilizes a range of proprietary platform technologies, including small molecule activators of innate immunity, phosphate-protected nucleotide prodrug chemistry, and glycoprotein-engineering of interferons. These technologies enable the development of distinct therapeutic products targeting serious viral conditions such as chronic hepatitis B and C, HIV, and respiratory viruses, including pandemic influenza and emerging diseases like SARS. Alios BioPharma's approach is supported by a proprietary chemical library of nucleoside analogs and advanced virology-based screening systems, positioning the company as a key player in the field of antiviral therapeutics.

Bird Rock Bio, Inc. is a clinical-stage biopharmaceutical company based in La Jolla, California, with a research facility in Shanghai, China. The company specializes in the discovery and development of novel biologic therapeutics aimed at addressing immune-inflammatory conditions, including rheumatoid arthritis, metabolic diseases, fibrosis, and nonalcoholic steatohepatitis. Bird Rock Bio's technology focuses on creating highly selective monoclonal antibodies targeting specific members of the G-protein coupled receptor (GPCR) family, a class of proteins critical for therapeutic efficacy. Its lead product, RYI-008, is a potent monoclonal antibody targeting IL-6, a cytokine linked to inflammation and cancer. The company is dedicated to meeting the medical needs of patients in China while also aiming for global impact. Bird Rock Bio has a growing pipeline of therapeutic antibodies developed through proprietary technologies that ensure specificity and effectiveness in treatment.

VHsquared Ltd. is a biotechnology company focused on developing oral biologics for immuno-inflammatory conditions affecting the gastrointestinal tract. Founded in 2010 and based in Cambridge, United Kingdom, VHsquared utilizes its proprietary Vorabody platform to create engineered domain antibodies that are resistant to degradation in the gut, allowing for effective oral administration. This innovative approach addresses the limitations of conventional antibodies, which are typically broken down in the gastrointestinal system. The company aims to produce a range of functional food products that target gastrointestinal infections and immune-mediated diseases, leveraging advancements in mucosal immunology and microbiota analysis. By developing therapies that are stable, specific, and locally active, VHsquared seeks to provide safer, more effective treatment options for patients with inflammatory bowel diseases and related conditions.

Aileron Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, dedicated to developing innovative therapeutics, particularly stabilized cell-permeating alpha-helical peptides, for oncology and other therapeutic areas. The company’s lead product candidate, ALRN-6924, is designed to treat advanced solid tumors and is currently undergoing various clinical trials, including Phase 2a studies for peripheral T-cell lymphoma and advanced solid tumors, as well as Phase 1 trials for acute myeloid leukemia and myelodysplastic syndromes. Aileron Therapeutics also explores next-generation wild type p53 reactivators and has established collaborations with notable institutions such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer to evaluate the effectiveness of ALRN-6924 in combination with other treatments. Founded in 2001 and originally known as Renegade Therapeutics, the company aims to address critical cell functions through its novel approach to modulating protein-protein interactions.

F-Star Therapeutics is a biopharmaceutical company focused on developing novel bispecific antibody products aimed at improving upon current standards of care, particularly in cancer therapy. The company is uniquely positioned with a strong patent portfolio, enabling it to create and develop Fcab antibody fragments and bispecific antibodies by modifying the constant region of an antibody. F-Star's Modular Antibody Technology allows for rapid discovery and development of bispecific antibodies by adding binding sites to the constant region, facilitating easier production compared to other antibody formats. The company is leveraging this technology to build a pipeline of product candidates. Founded in 2006, F-Star has received significant funding from leading venture capital firms and strategic corporate investors. The company operates out of Cambridge, UK, with over 35 employees dedicated to research and development.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

eFFECTOR Therapeutics, Inc. is a clinical-stage biotechnology company based in San Diego, California, that specializes in the development of selective translation regulators for the treatment of cancer and other serious diseases. Founded in 2012, the company is pioneering a new class of oncology drugs known as selective translation regulator inhibitors (STRIs). These drug candidates specifically target the eIF4F complex and its associated kinase, MNK 1/2, which play critical roles in protein synthesis and are linked to prominent cancer signaling pathways such as PI3K-AKT and RAS-MEK. eFFECTOR's products aim to inhibit pathways that drive tumor growth, survival, and immune evasion by regulating the expression of oncoproteins and immunosuppressive proteins. The lead candidate, tomivosertib, is currently being evaluated in a Phase 2b trial for non-small cell lung cancer in combination with pembrolizumab. Additionally, zotatifin, an inhibitor of eIF4A, is undergoing dose-escalation in a Phase 1/2 trial, with further expansion cohorts anticipated. The company also collaborates with Pfizer to advance inhibitors targeting eIF4E.

Aileron Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, dedicated to developing innovative therapeutics, particularly stabilized cell-permeating alpha-helical peptides, for oncology and other therapeutic areas. The company’s lead product candidate, ALRN-6924, is designed to treat advanced solid tumors and is currently undergoing various clinical trials, including Phase 2a studies for peripheral T-cell lymphoma and advanced solid tumors, as well as Phase 1 trials for acute myeloid leukemia and myelodysplastic syndromes. Aileron Therapeutics also explores next-generation wild type p53 reactivators and has established collaborations with notable institutions such as Dana-Farber/Boston Children’s Cancer and Blood Disorders Center and Pfizer to evaluate the effectiveness of ALRN-6924 in combination with other treatments. Founded in 2001 and originally known as Renegade Therapeutics, the company aims to address critical cell functions through its novel approach to modulating protein-protein interactions.

Bicycle Therapeutics plc is a clinical-stage biopharmaceutical company based in Cambridge, United Kingdom, specializing in the development of a novel class of medicines known as Bicycles. These are synthetic short peptides designed to form two loops that stabilize their structure. The company's lead product candidate, BT1718, is a Bicycle Toxin Conjugate (BTC) currently undergoing Phase I/IIa clinical trials targeting tumors that express Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also a BTC in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies targeting Nectin-4. Additionally, Bicycle is advancing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to address therapeutic areas such as anti-infective, cardiovascular, ophthalmology, and respiratory indications. Bicycle Therapeutics was incorporated in 2009 and aims to address significant unmet medical needs in oncology and beyond.

Applied Genetic Technologies Corporation is a clinical-stage biotechnology company focused on developing innovative genetic therapies for patients with rare and debilitating diseases. The company has advanced product candidates in ophthalmology, including treatments for X-linked retinitis pigmentosa and achromatopsia, with the former having completed Phase I/II trials and the latter currently in the same stage. Additionally, AGTC is working on an optogenetic therapy for patients with advanced retinal diseases. Beyond ophthalmology, the company has initiated preclinical programs targeting otology and central nervous system disorders, including frontotemporal dementia, amyotrophic lateral sclerosis, and adrenoleukodystrophy. AGTC employs gene therapy techniques to replace defective genes with functional ones, aiming to provide long-lasting treatment solutions through single injections. Established in 1999 and headquartered in Alachua, Florida, the company collaborates with various academic and industry partners to enhance its research and development efforts.

Principia Biopharma Inc. is a late-stage biopharmaceutical company headquartered in South San Francisco, California, focused on developing innovative therapies for immune-mediated diseases. The company's lead product, rilzabrutinib, is an inhibitor undergoing Phase III clinical trials for pemphigus and pemphigus foliaceus, as well as Phase 1/2 trials for immune thrombocytopenia and Phase 2 trials for IgG4-related disease. Additionally, Principia is developing PRN2246/SAR442168, which is in Phase II for multiple sclerosis and other central nervous system conditions, alongside PRN473, a drug candidate in Phase I trials for immune-mediated diseases. Principia Biopharma maintains a collaboration agreement with Genzyme Corporation for the development of treatments for MS and other CNS diseases. Originally incorporated in 2008, the company is dedicated to addressing significant unmet medical needs in immunology and oncology through its innovative drug development platform.

Genocea Biosciences, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, focused on the discovery and development of innovative cancer immunotherapies and vaccines. Utilizing its proprietary ATLAS platform, the company profiles patient-specific CD4+ and CD8+ T cell immune responses to identify potential antigens in tumors. This technology enables Genocea to develop personalized immuno-oncology programs, including GEN-009, an adjuvanted neoantigen peptide vaccine currently in phase I/IIa clinical trials, as well as GEN-011, a neoantigen adoptive T cell therapy, and GEN-010, a neoantigen vaccine program. Additionally, the company has pursued immunotherapies for other diseases, such as GEN-003, aimed at treating genital herpes, and it maintains a pre-clinical program focused on personalized cancer vaccines. Founded in 2006, Genocea is dedicated to addressing significant unmet medical needs through its advanced therapeutic approaches.

IlluminOss Medical, Inc., established in 2007 and headquartered in East Providence, Rhode Island, is a medical device company specializing in orthopedic fracture repair. It designs, develops, and markets products based on its proprietary IlluminOss System, which employs a light-curable polymer within an expandable balloon catheter to create patient-specific intramedullary implants for bone stabilization. This minimally invasive technology is particularly suited for treating osteoporotic and compromised bone. The company's products are CE-marked and FDA-cleared for various anatomical sites and are distributed in several European countries.

Akamis Bio is a clinical-stage oncology company dedicated to advancing cancer treatment through its innovative Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform. The company develops a portfolio of therapeutics specifically targeting solid tumors, aiming to enhance patients' immune responses to recognize and eradicate cancer cells. By harnessing a proprietary intravenously administered viral platform, Akamis Bio focuses on delivering effective gene therapies that address the needs of cancer patients, striving to improve their quality of life and treatment outcomes.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Nimbus Therapeutics, LLC is a biotechnology company based in Cambridge, Massachusetts, focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. Founded in 2009, the company utilizes a computational technology-driven approach to advance a pipeline of novel small-molecule therapeutics. Nimbus Therapeutics specializes in designing allosteric inhibitors, notably targeting Acetyl-CoA Carboxylase for conditions such as non-alcoholic steatohepatitis and hepatocellular carcinoma. The company has established strategic partnerships with organizations like Charles River Laboratories and Celgene Corporation to enhance its drug development efforts. In March 2015, Nimbus Discovery rebranded itself as Nimbus Therapeutics to better reflect its transition into a clinical-stage enterprise.

Constellation Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on developing innovative therapeutics for unmet medical needs in cancer treatment. The company specializes in the field of epigenetics, aiming to address cancers associated with abnormal gene expression and drug resistance. Its lead product candidates include CPI-0610, which is undergoing Phase II clinical trials for myelofibrosis in combination with ruxolitinib, and CPI-1205, currently in Phase Ib/II trials for metastatic castration-resistant prostate cancer in conjunction with androgen receptor signaling inhibitors. Additionally, Constellation is developing CPI-0209, which is in the Phase I dose escalation stage of a Phase I/II trial aimed at treating solid tumors. Founded in 2008, the company was previously known as Epigenetix, Inc. before rebranding to its current name in March 2008.