RA Capital Management

Imbria Pharmaceuticals, Inc. is a clinical-stage pharmaceuticals company based in Boston, Massachusetts, founded in 2018. The company specializes in developing innovative therapies aimed at addressing cardiometabolic disorders by enhancing or restoring cellular energy production. Imbria's research focuses on the mechanisms underlying these conditions, particularly in areas such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction. By translating scientific insights into practical treatments, Imbria Pharmaceuticals aims to meet significant unmet medical needs and improve the lives of patients affected by these life-altering disorders. Through its commitment to understanding cellular metabolism and mitochondrial function, the company works to advance healthcare solutions in the cardiometabolic space.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Elektrofi is a preclinical-stage company based in Boston, Massachusetts, focused on advancing drug delivery methods to enhance patient experiences. Established in 2016, the company has developed a formulation system capable of processing a diverse array of therapeutic molecules, including large and complex proteins such as monoclonal antibodies, bispecifics, and fusion proteins. Its innovative technology aims to improve subcutaneous administration, allowing drug manufacturers to create breakthrough therapies that empower patients with more control over how they receive essential medications.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to create targeted treatments that address critical cytokine signaling pathways involved in these diseases. By integrating insights from disease mechanisms, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to design precise therapeutics that meet significant unmet medical needs for patients suffering from hematologic malignancies.

ARTARA THERAPEUTICS is a clinical-stage healthcare company focused on developing innovative therapies for rare and specialty diseases. Its leading program, TARA-002, is an investigational cell therapy aimed at treating lymphatic malformations and non-muscle invasive bladder cancer. Additionally, the company is advancing intravenous choline chloride, a phospholipid substrate replacement therapy designed for patients suffering from intestinal failure-associated liver disease. Based in New York, ARTARA THERAPEUTICS is committed to addressing significant unmet medical needs by bringing transformative therapies to patients with limited treatment options.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, founded in 2014. It specializes in developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. The company utilizes a synthetic amplifier technology that enhances the speed and complexity of antibiotic susceptibility testing, facilitating quicker and more accurate selection of personalized antimicrobial therapies for patients. By streamlining the identification of effective treatments, SeLux Diagnostics aims to improve patient care, reduce hospital stays, and tackle the growing issue of antibiotic resistance by minimizing the reliance on broad-spectrum antibiotics.

Astria Therapeutics is a biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing innovative therapies for rare and niche allergic and immunological diseases. The company focuses on bringing life-changing treatments to patients and families affected by these conditions. Its lead program, STAR-0215, is a monoclonal antibody that inhibits plasma kallikrein and is currently in preclinical development for hereditary angioedema. Additionally, Astria is developing STAR-0310, a monoclonal antibody OX40 antagonist, which is also in preclinical stages and targets atopic dermatitis, an immune disorder characterized by compromised skin barrier function and associated itching.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

AM Batteries manufactures Li-ion batteries using solvent-free electrode manufacturing technology. The company was founded in 2016 and is headquartered in Chelmsford, Massachusetts.

SAB Biotherapeutics, Inc. is a clinical-stage biopharmaceutical development company based in Sioux Falls, South Dakota, with an additional office in Cambridge, Massachusetts. Founded in 2014, the company focuses on developing immunotherapies for various human diseases using its proprietary DiversitAb platform. This innovative platform enables the rapid production of fully targeted human polyclonal antibodies from genetically engineered transchromosomic cattle, eliminating the need for human plasma or serum. SAB Biotherapeutics' product pipeline includes SAB-142 for Type 1 diabetes, SAB-176 for seasonal influenza, and SAB-301 for Middle East Respiratory Syndrome Coronavirus (MERS-CoV). The company's approach aims to address public health challenges, rare diseases, and pandemic threats through advanced antibody science.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.

Vaxess Technologies, Inc. is a biotechnology company based in Boston, Massachusetts, founded in 2011. The company focuses on developing and commercializing innovative silk biomaterial technologies. Its key product, the MIMIX microneedle platform, facilitates the transdermal delivery of vaccines and therapeutics. This sustained release patch technology allows for a controlled release of vaccines, mimicking the natural infection process to enhance immune response and effectiveness. Vaxess's patch is engineered for stability, eliminating the need for refrigeration, which enables shipping and application in low-resource settings. In addition to its vaccine delivery systems, Vaxess is also engaged in creating novel therapies aimed at oncology care and infectious diseases, ultimately striving to improve patient outcomes with reduced treatment burdens.

Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Empatica Inc. is a company that specializes in developing wearable medical sensor devices aimed at health monitoring, treatment, and research. Founded in 2013 and based in Cambridge, Massachusetts, Empatica's offerings include the E3 wristband, which monitors physiological signals in real-time, along with a mobile application and a web dashboard designed for stress and sleep monitoring. The company employs a combination of biosensors and artificial intelligence to analyze human physiology, providing valuable insights for patients, clinicians, and researchers. Its client base includes notable organizations such as Microsoft Research, Sony, University of Trento, and Intel.

Concert Pharmaceuticals is a clinical-stage biopharmaceutical company focused on discovering and developing novel small molecule drugs for autoimmune and central nervous system disorders. The company employs an innovative precision deuterium chemistry platform to enhance the properties of established drug molecules, which enables the creation of new chemical entities with potential efficacy and safety advantages. Notable product candidates include CTP-543, currently in Phase III trials for alopecia areata, CTP-692 for schizophrenia in Phase II trials, and AVP-786, which targets neurological and psychiatric disorders in Phase III trials. Concert Pharmaceuticals also engages in strategic collaborations with various pharmaceutical companies to support its development efforts. Founded in 2006 and based in Lexington, Massachusetts, the company aims to streamline research and development processes, reducing associated risks and costs while advancing its therapeutic pipeline.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, founded in 2014. It specializes in developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. The company utilizes a synthetic amplifier technology that enhances the speed and complexity of antibiotic susceptibility testing, facilitating quicker and more accurate selection of personalized antimicrobial therapies for patients. By streamlining the identification of effective treatments, SeLux Diagnostics aims to improve patient care, reduce hospital stays, and tackle the growing issue of antibiotic resistance by minimizing the reliance on broad-spectrum antibiotics.

Cedilla is a developer of therapeutics technologies intended to broaden the reach of small molecule therapeutics. The company's technology is focused on destabilizing oncoprotein targets that are also applicable to therapies outside of oncology, enabling doctors to treat diseases caused by protein dysregulation. The company was founded in 2018 and based in Cambridge, Massachusetts.

Delix Therapeutics is a neuroscience company focused on harnessing the power of psychoplastogens—novel neuroplasticity-promoting therapeutics—to better treat mental health disorders at scale. The company was founded in 2019 and is headquartered in Boston, Massachusetts.

HilleVax is a biopharmaceutical company that focuses on the development and commercialization of novel vaccines. HilleVax was founded in 2021 and was headquartered in Boston, Massachusetts.

Jnana Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that focuses on drug discovery through its innovative platform targeting solute carrier (SLC) transporters. Established in 2016, the company aims to improve patient outcomes by developing small molecule therapies for various diseases, including those related to immunometabolism, lysosomal function, and mucosal defense. By utilizing advanced chemoproteomics techniques, Jnana accelerates the identification of therapeutic targets, addressing significant unmet medical needs in areas such as immuno-oncology, inflammatory disorders, and neurological diseases. The company's approach seeks to enhance drug discovery efficiency by comprehensively targeting the vast family of membrane proteins associated with SLC transporters.

Tango Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative cancer therapies through the principle of synthetic lethality. Established in 2014, the company aims to discover and target specific driver genes involved in cancer progression. Its research concentrates on three main areas: counteracting the loss of tumor suppressor genes, reversing cancer cells' ability to evade the immune system, and identifying effective drug combinations that outperform single-agent therapies. Tango's pipeline includes TNG908, a selective inhibitor of PRMT5 designed for cancer cells with an MTAP deletion, alongside other discovery programs like TNG462, TNG348, and TNG260, which target various cancer types with limited treatment options.

GentiBio, Inc. is a biotherapeutics company focused on developing engineered regulatory T cells (EngTregs) aimed at treating autoimmune, alloimmune, autoinflammatory, and allergic diseases. Founded in 2020 and headquartered in Boston, Massachusetts, with additional locations in Israel and Seattle, GentiBio utilizes a proprietary platform that combines autologous and allogeneic EngTregs technologies. This innovative approach is designed to restore immune tolerance and address limitations present in current regulatory T-cell therapies. By targeting the underlying causes of diseases related to immune system dysfunction, GentiBio aims to provide effective therapeutic solutions for conditions that arise from immune system overshooting or malfunctioning. The company was co-founded by experts in Treg biology and synthetic immunology, positioning it at the forefront of this emerging field.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.

Century Therapeutics is a biotechnology company focused on developing advanced cell therapies using induced pluripotent stem cells (iPSCs) to treat various cancers, including hematologic and solid tumors. Founded in 2018 and based in Philadelphia, Pennsylvania, the company utilizes a unique allogeneic iPSC-derived cell therapy platform that integrates gene editing, protein engineering, and robust manufacturing capabilities. This innovative approach allows for the generation of modified immune effector cells, such as natural killer (NK) and T cells, designed to meet significant unmet medical needs. By employing techniques like CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to enhance cell product performance and minimize rejection by the host immune system, thereby improving the efficacy of cancer treatments.

Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies that leverage its proprietary technology to maximize the effectiveness of existing immuno-oncology treatments while minimizing side effects outside the tumor. Its lead product, XTX201, is designed to induce immune activity specifically within tumors, demonstrating improved antitumor efficacy. Xilio Therapeutics aims to deliver significant improvements in patient outcomes by utilizing its platform to create a pipeline of novel therapies, including tumor-activated cytokines, antibodies, and immune cell engagers, all targeted to optimize therapeutic effects within the tumor microenvironment. Founded in 2015 and based in Waltham, Massachusetts, the company was previously known as Akrevia Therapeutics before rebranding in March 2020.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company based in Lexington, Massachusetts, focused on developing innovative therapies that leverage the biology of DNA repair for cancer treatment and synthetic lethality in autoimmune diseases. The company utilizes its expertise in DNA damage response biology to create a pipeline of small molecule therapeutics aimed at selectively targeting vulnerabilities in cancer cells and inducing their self-destruction through DNA damage overload. Cyteir's lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, which is crucial for repairing double-strand DNA breaks. The company is dedicated to addressing a variety of hematological malignancies, solid tumors, and chronic autoimmune disorders through its integrated drug development platform. Founded in 2012, Cyteir is committed to advancing next-generation therapies that can improve patient outcomes in challenging disease states.

TScan Therapeutics is a biopharmaceutical company dedicated to discovering and developing innovative T cell receptor (TCR) engineered therapies to combat various cancers. Based in Waltham, Massachusetts, the company focuses on TCR-T therapies for both liquid cancers and solid tumors. Its lead candidates, TSC-100 and TSC-101, are designed to address hematologic malignancies, specifically targeting residual leukemia and preventing relapse after hematopoietic stem cell transplantation. In addition, TScan is advancing a multiplexed TCR-T therapy program aimed at treating a range of solid tumors. The company's proprietary platform identifies clinically relevant T cell antigens and minimizes off-target effects, enhancing the precision of its therapies. Founded in 2018, TScan Therapeutics is committed to improving outcomes for patients with serious diseases.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Based in Newton, Massachusetts, Abcuro creates therapeutic antibodies targeting novel mechanisms related to these diseases. The company utilizes advanced bioinformatics to analyze transcription data from human diseases, which aids in the identification of new therapeutic targets. Through ex-vivo validation in diseased tissues, Abcuro seeks to uncover innovative methods to modulate key components of the immune system. Established in 2015, Abcuro aims to enhance treatment strategies in the healthcare industry by offering effective solutions for complex immunological conditions.

Werewolf Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapeutics that enhance the body’s immune response to cancer. Founded in 2017, the company utilizes its proprietary PREDATOR platform to engineer conditionally activated molecules, known as INDUKINE molecules. These therapeutics are designed to remain inactive in peripheral tissues but activate selectively within the tumor microenvironment, addressing the limitations of traditional proinflammatory immune therapies. By stimulating both adaptive and innate immunity, Werewolf Therapeutics aims to improve cancer treatment outcomes.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Be Biopharma, Inc. is focused on developing innovative therapeutics derived from engineered B-cells to address various diseases, including cancer, autoimmune disorders, and infectious diseases. Founded in 2020 and based in Cambridge, Massachusetts, the company aims to overcome significant challenges associated with traditional cell and gene therapies, such as the limitations of dosing, unpredictable reactions in patients, and the necessity of pre-treatment chemotherapy. By leveraging precision genome editing, Be Biopharma creates durable and effective cell therapies that can be administered without toxic conditioning, thereby improving treatment options for conditions like Hemophilia B and enhancing overall patient outcomes.

Nimbus Therapeutics, based in Cambridge, Massachusetts, is a biotechnology company focused on discovering and developing innovative medicines for serious diseases, including metabolic disorders, cancer, and immune-inflammatory conditions. The company utilizes advanced computational technologies to create selective and potent small-molecule therapeutics. Among its notable projects is the Nimbus Apollo program, which features the lead candidate NDI-010976, an allosteric inhibitor targeting Acetyl-CoA Carboxylase (ACC). This compound is being developed for the treatment of non-alcoholic steatohepatitis (NASH) and has potential applications in hepatocellular carcinoma (HCC) and other diseases. NDI-010976 received Fast Track designation from the U.S. Food and Drug Administration in 2016, highlighting its significance in the clinical development pipeline. Nimbus Therapeutics has formed strategic partnerships to enhance its research and development capabilities, reinforcing its commitment to advancing treatment options for complex health challenges.

PMV Pharmaceuticals, Inc. is a precision oncology company focused on discovering and developing small molecule therapies targeting p53 mutations in cancer. The company's lead candidate, PC14586, aims to correct and restore the function of p53, a critical tumor suppressor protein. In addition to this product, PMV Pharmaceuticals is also working on therapies for specific p53 hotspot mutations, including the R273H mutation. Founded in 2013 and headquartered in Cranbury, New Jersey, the company leverages over three decades of expertise in p53 biology, aiming to develop first-in-class modulators that could improve treatment outcomes for cancer patients.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Established in 2018 and based in Cambridge, Massachusetts, the company is advancing a diversified pipeline that includes several clinical-stage investigational therapies targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Notable products in development include CVL-231, a positive allosteric modulator currently in Phase Ib trials for schizophrenia, and CVL-865, also a positive allosteric modulator, which is in Phase II trials for drug-resistant focal onset seizures. Additionally, Tavapadon, a selective dopamine D1/D5 partial agonist, is in a Phase III program for Parkinson's disease, while CVL-936, a selective dopamine D3-preferring antagonist, is undergoing Phase I trials for substance use disorder. The company is also engaged in preclinical research aiming to address various neuroscience indications, leveraging expertise in neurocircuitry and receptor selectivity.

Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that focuses on discovering and developing antiviral therapies for severe viral infections. The company's lead candidate, AT-527, is an antiviral agent currently undergoing phase 3 clinical trials for the treatment of COVID-19, caused by SARS-CoV-2. Additionally, Atea is developing several other product candidates, including AT-787, which is in phase 2 trials for hepatitis C, and AT-752 for dengue. Other candidates, such as AT-889 and AT-934, are also in phase 2 clinical trials targeting respiratory syncytial virus. Established in 2014, Atea Pharmaceuticals aims to improve treatment options for patients suffering from serious viral infections.

Pandion Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapeutics for patients with autoimmune diseases and inflammatory conditions. The company's lead product candidate, PT101, is an engineered variant of interleukin-2 currently undergoing Phase 1a clinical trials for 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 PT001 and PT002, which are bifunctional molecules designed for tissue-selective immunomodulation in the gastrointestinal tract. These molecules utilize a unique approach to achieve localized treatment, potentially enhancing efficacy and safety compared to traditional therapies. Pandion also collaborates with Astellas Pharma to develop locally acting immunomodulators targeting autoimmune diseases of the pancreas. The company is headquartered in Watertown, Massachusetts.

Akouos, Inc. is a biotechnology company based in Boston, Massachusetts, dedicated to developing gene therapies aimed at restoring, improving, and preserving hearing for individuals affected by various inner ear disorders. Founded in 2016, the company utilizes an innovative platform that includes a proprietary adeno-associated viral vector library and a unique delivery method. Its leading product candidate, AK-OTOF, targets hearing loss resulting from mutations in the OTOF gene. Akouos focuses on genetically-defined patient populations, addressing conditions ranging from single genetic mutations to hearing loss caused by ototoxic drugs and aging. The company has established strategic partnerships with key institutions, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing precision genetic therapies for sensorineural hearing loss.

Quench Bio, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing therapies for inflammatory diseases through the exploration of gasdermin biology and innate immunity. The company’s lead compounds specifically target and inhibit Gasdermin D, a protein involved in the processes of pyroptosis and NETosis, which play a crucial role in the release of inflammatory cytokines, alarmins, DNA, and neutrophil extracellular traps (NETs). By addressing these pathways, Quench Bio aims to provide relief for patients suffering from severe inflammatory conditions. Originally founded as Project Pyro, the company changed its name to Quench Bio in June 2018.

Covid Apollo is a venture capital firm based in Boston, Massachusetts, founded in 2020. The firm focuses on investing in the biotechnology and life science sectors, specifically targeting solutions related to COVID-19 testing. By combining investment funds with expertise in diagnostics, Covid Apollo aims to identify, scale, and bring innovative COVID-19 testing solutions to market.

Aerovate Therapeutics is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative treatments for patients with rare cardiopulmonary diseases. The company's primary focus is on AV-101, a proprietary inhaled dry powder formulation of the drug imatinib, designed specifically for the treatment of pulmonary arterial hypertension (PAH). This formulation enables targeted delivery of the medication directly to affected lung tissues, thereby minimizing systemic side effects commonly associated with traditional treatments. Established in 2018, Aerovate aims to provide meaningful improvements in the lives of those suffering from these serious conditions.

FORMA Therapeutics is a clinical-stage biopharmaceutical company based in Watertown, Massachusetts, specializing in the development and commercialization of innovative therapeutics for rare hematologic diseases and various cancers. The company's notable product candidates include FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, which targets metastatic castration-resistant prostate cancer. Additionally, FORMA is advancing FT-2102, an oral investigational agent designed to inhibit mutated IDH1 enzymes, now being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and exploratory Phase 1 trials for glioma. The company also develops FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. By integrating advanced drug discovery technologies and oncology expertise, FORMA aims to target challenging biological pathways that contribute to cancer, thereby enhancing the development of small molecule drug candidates with unique mechanisms of action.

Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing and commercializing therapeutics for rare genetic disorders of hemoglobin, including sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing its lead product candidate, IMR-687, which is a highly selective small molecule inhibitor of PDE9. IMR-687 is designed as an oral, once-a-day treatment that aims to modify disease through a multimodal mechanism of action affecting various cell types, including red and white blood cells. Imara is dedicated to addressing the unmet medical needs of patients suffering from these serious hemoglobinopathies.

Eliem Therapeutics, Inc. is a clinical-stage biotechnology company based in Boston, Massachusetts, dedicated to developing innovative therapeutic drugs for hyperexcitability disorders affecting the nervous system. Founded in 2018, the company focuses on creating treatments for conditions such as chronic pain, depression, epilepsy, and anxiety, which significantly impact the quality of life for millions. Eliem employs its expertise in neuroscience and translational medicine to advance a promising pipeline of drug candidates, aiming to address unmet medical needs in these areas. Notably, its leading drug candidate, ETX-155, targets major depressive disorder and focal onset seizures. Through its efforts, Eliem Therapeutics seeks to improve the lives of patients suffering from these debilitating disorders.

Vor Biopharma, Inc. is a clinical-stage biotechnology company based in Cambridge, Massachusetts, focused on developing engineered hematopoietic stem cell (eHSC) therapies for cancer treatment. The company's lead product candidate, VOR33, is designed specifically for the treatment of acute myeloid leukemia (AML) and is currently in preclinical development. VOR33 eHSCs are engineered to lack CD33, a protein commonly expressed by AML cells, allowing for selective targeting of the cancer without harming normal cells. This innovative approach seeks to minimize the toxicities associated with traditional immunotherapies, which often affect both cancerous and healthy tissues. By protecting healthy stem cells from depletion while generating functional immune cells, Vor Biopharma aims to enhance therapeutic outcomes for patients with hematological malignancies.

Black Diamond Therapeutics is a precision oncology medicine company focused on discovering and developing small molecule therapies that are tumor-agnostic. The company’s lead product candidate, BDTX-189, targets non-canonical and oncogenic mutations in ErbB kinases, specifically the epidermal growth factor receptor and tyrosine-protein kinase. In addition to BDTX-189, Black Diamond is developing allosteric-EGFR mutation inhibitors and other early-stage pipeline programs that address allosteric mutations in kinases associated with cancer and rare genetic diseases. The company's approach is centered on its Mutation-Allostery-Pharmacology platform, which aims to create targeted treatments for genetically defined cancers, especially for patients with limited existing treatment options. Founded in 2014 and headquartered in Cambridge, Massachusetts, Black Diamond Therapeutics was previously known as ASET Therapeutics until its name change in January 2018.

SeLux Diagnostics, Inc. is a biotechnology company based in Charlestown, Massachusetts, founded in 2014. It specializes in developing a next-generation phenotyping platform for antibiotic susceptibility testing and diagnostics of infectious diseases. The company utilizes a synthetic amplifier technology that enhances the speed and complexity of antibiotic susceptibility testing, facilitating quicker and more accurate selection of personalized antimicrobial therapies for patients. By streamlining the identification of effective treatments, SeLux Diagnostics aims to improve patient care, reduce hospital stays, and tackle the growing issue of antibiotic resistance by minimizing the reliance on broad-spectrum antibiotics.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

Imbria Pharmaceuticals, Inc. is a clinical-stage pharmaceuticals company based in Boston, Massachusetts, founded in 2018. The company specializes in developing innovative therapies aimed at addressing cardiometabolic disorders by enhancing or restoring cellular energy production. Imbria's research focuses on the mechanisms underlying these conditions, particularly in areas such as non-obstructive hypertrophic cardiomyopathy, stable angina, and heart failure with preserved ejection fraction. By translating scientific insights into practical treatments, Imbria Pharmaceuticals aims to meet significant unmet medical needs and improve the lives of patients affected by these life-altering disorders. Through its commitment to understanding cellular metabolism and mitochondrial function, the company works to advance healthcare solutions in the cardiometabolic space.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of therapies designed to degrade disease-causing proteins. The company’s lead product candidates include ARV-110, a proteolysis-targeting chimera (PROTAC) currently in phase I clinical trials for treating metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. Additionally, Arvinas is developing other PROTACs aimed at degrading various androgen receptor mutations and has initiatives focused on treating neurodegenerative diseases, including tauopathies. Arvinas collaborates with major pharmaceutical companies such as Pfizer, Genentech, Roche, and Bayer to advance its innovative therapeutic approaches. Founded in 2015, Arvinas is dedicated to improving the lives of patients with debilitating and life-threatening conditions through its unique protein degradation technology.

Lantos Technologies, Inc. specializes in 3D digital ear scanning technology, which facilitates the design of personalized in-ear devices such as headphones, musicians' monitors, hearing aids, and noise protection solutions. The company's AURA 3D ear scanning system is the only FDA-cleared solution of its kind, providing accurate and minimally invasive mapping of the ear canal. This technology collects multiple data points to create detailed digital maps of the ear, improving the comfort, sound quality, and performance of custom products. By enhancing clinical efficiency and enabling faster turnaround times for custom devices, Lantos Technologies aims to deliver better outcomes for patients and audiologists alike. Founded in 2009 and based in Derry, New Hampshire, the company was previously known as Lantos 2.0 Inc. before rebranding in December 2020.

Prevail Therapeutics is a biotechnology company focused on developing novel gene therapies for patients with Parkinson’s disease and other neurodegenerative diseases. Prevail was launched in 2017 by The Silverstein Foundation for Parkinson’s with GBA, REGENXBIO, and OrbiMed, and is headquartered in New York, NY.

Dyne Therapeutics is a biotechnology company based in Waltham, Massachusetts, dedicated to developing targeted therapies for genetically driven muscle diseases. Founded in 2017, the company focuses on advancing treatments for conditions such as myotonic dystrophy type 1, Duchenne muscular dystrophy, and facioscapulohumeral dystrophy, among others. Utilizing its innovative FORCE platform, Dyne Therapeutics aims to deliver disease-modifying therapies with enhanced precision to skeletal, cardiac, and smooth muscle tissues. The company's lead product candidates, DYNE-101 and DYNE-251, are currently undergoing phase 1/2 clinical trials, reflecting its commitment to addressing the unmet medical needs of patients with serious muscle disorders.

TARIS Biomedical, Inc. is a therapeutically focused urology company based in Lexington, Massachusetts, established in 2008. The company specializes in developing innovative treatments for challenging bladder diseases, including bladder cancer and overactive bladder. TARIS's unique drug delivery system allows for the continuous release of medications within the bladder over extended periods, ranging from weeks to months. This system is designed to be deployed and retrieved through minimally invasive, in-office procedures, enabling a tailored approach to drug release that meets the specific needs of various bladder conditions. As of December 2019, TARIS Biomedical operates as a subsidiary of Janssen Research & Development, LLC.

Kadmon is a biopharmaceutical company focused on discovering, developing, and commercializing small molecules and biologics for the treatment of inflammatory and fibrotic diseases. The company’s lead product candidates include KD025, an oral selective inhibitor targeting rho-associated coiled-coil kinase 2, currently in Phase II clinical trials for chronic graft-versus-host disease and fibrotic conditions. Other significant candidates are KD045, also an oral inhibitor for fibrotic diseases, and KD033, an anti-PD-L1/IL-15 fusion protein aimed at cancer treatment. Kadmon is also developing Tesevatinib for autosomal dominant polycystic kidney disease and provides ribavirin for hepatitis treatment. The company offers patient assistance programs and other support services to enhance treatment access and adherence. Founded in 2010 and headquartered in New York, Kadmon has established strategic collaborations and license agreements to further its research and development efforts.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

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. The company’s leading product candidate, tebipenem pivoxil hydrobromide, is an oral antibiotic designed to treat MDR Gram-negative infections in adults. Additionally, Spero is advancing SPR206, an intravenous agent targeting MDR Gram-negative infections, and SPR720, an oral antibiotic aimed at treating pulmonary non-tuberculous mycobacterial disease. Spero Therapeutics collaborates with various organizations, including Meiji Seika Pharma for tebipenem HBr, Everest Medicines for SPR206 in Asia, and the Bill & Melinda Gates Medical Research Institute for SPR720. Founded in 2013, Spero leverages a strong team in chemistry and microbiology to address significant unmet medical needs in infectious disease treatment.

Bristol-Myers Squibb Company is a global biopharmaceutical firm engaged in the discovery, development, licensing, manufacturing, and marketing of pharmaceutical products. The company specializes in therapeutics across several key areas, including hematology, oncology, cardiovascular health, and immunology. Notable products include Opdivo for cancer treatment, Eliquis for stroke prevention and venous thromboembolic disorders, and Orencia for rheumatoid arthritis. Additionally, Bristol-Myers Squibb offers therapies for chronic hepatitis B and HIV, among others. The company has significant collaborations with various partners in the pharmaceutical industry, including major players like Pfizer and AstraZeneca. Founded in 1887 and headquartered in New York City, Bristol-Myers Squibb has a strong presence in the U.S. market, which accounts for approximately 70% of its total sales.

Intarcia Therapeutics, Inc. is a biopharmaceutical company focused on developing therapies for chronic conditions that require long-term management. The company is known for its innovative Medici Drug Delivery System, which facilitates subcutaneous delivery of therapies for diseases such as type 2 diabetes, obesity, and autoimmune disorders. Its lead product, ITCA 650, is currently in phase 3 clinical trials designed to provide sustained treatment for diabetes through a steady-state dosing system. Additionally, Intarcia's DUROS platform stabilizes and delivers therapeutic proteins and peptides, including devices aimed at treating conditions such as Hepatitis C. Founded in 1997 and originally named BioMedicines, the company rebranded to Intarcia Therapeutics in 2004. It is headquartered in Boston, Massachusetts, with a manufacturing facility in Hayward, California, and a research and development center in Durham, North Carolina.

Antera Therapeutics is a biotechnology company that specializes in creating therapies that will prevent allergy development in children. It helps families introduce peanuts into infants'​ diets in a structured and convenient way. It was founded in 2014 and headquartered in Boston, Massachusetts.

Kala Pharmaceuticals, Inc. is a biopharmaceutical company headquartered in Watertown, Massachusetts, that specializes in developing therapies for diseases affecting mucosal tissues using its proprietary nanoparticle-based Mucus Penetrating Particles (MPP) technology. This innovative approach enhances drug distribution and pharmacokinetics by delivering biocompatible, drug-loaded particles that uniformly coat mucosal surfaces. The company's lead product candidate, KPI-121 0.25%, has successfully completed two Phase III clinical trials aimed at providing temporary relief for dry eye disease. Additionally, INVELTYS, another product, has also completed two Phase III trials for treating inflammation and pain following ocular surgery. Kala is advancing its pipeline, including KPI-285, a receptor tyrosine kinase inhibitor for retinal diseases, which is currently in preclinical studies. The company aims to address significant clinical needs while exploring research collaborations to enhance its product offerings.

Adeo Health Science, Inc. is a Boston-based company that develops and produces organic foods specifically designed for special dietary needs. Its flagship product, Aralyte, incorporates natural distilled peanut extract and Vitamin D, providing a structured approach for parents to implement the LEAP regimen for preventing peanut allergies in infants starting solid food. Founded in 2014, the company is committed to translating cutting-edge research on food allergy prevention into accessible and reliable products for families. Adeo Health Science offers its products for sale online, reflecting its focus on both innovation and consumer accessibility.

TARIS Biomedical, Inc. is a therapeutically focused urology company based in Lexington, Massachusetts, established in 2008. The company specializes in developing innovative treatments for challenging bladder diseases, including bladder cancer and overactive bladder. TARIS's unique drug delivery system allows for the continuous release of medications within the bladder over extended periods, ranging from weeks to months. This system is designed to be deployed and retrieved through minimally invasive, in-office procedures, enabling a tailored approach to drug release that meets the specific needs of various bladder conditions. As of December 2019, TARIS Biomedical operates as a subsidiary of Janssen Research & Development, LLC.

Arvinas, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, that specializes in the discovery, development, and commercialization of therapies designed to degrade disease-causing proteins. The company’s lead product candidates include ARV-110, a proteolysis-targeting chimera (PROTAC) currently in phase I clinical trials for treating metastatic castration-resistant prostate cancer, and ARV-471, which targets the estrogen receptor for patients with metastatic ER-positive/HER2-negative breast cancer. Additionally, Arvinas is developing other PROTACs aimed at degrading various androgen receptor mutations and has initiatives focused on treating neurodegenerative diseases, including tauopathies. Arvinas collaborates with major pharmaceutical companies such as Pfizer, Genentech, Roche, and Bayer to advance its innovative therapeutic approaches. Founded in 2015, Arvinas is dedicated to improving the lives of patients with debilitating and life-threatening conditions through its unique protein degradation technology.

Ra Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company headquartered in Cambridge, Massachusetts, focused on developing therapeutics for diseases associated with the overactivation of the complement system. Utilizing a peptide chemistry platform, the company creates synthetic macrocyclic peptides that combine the specificity of antibodies with the pharmacological advantages of small molecules. Its lead candidate, Zilucoplan, is an injectable treatment that has completed Phase II clinical trials for generalized myasthenia gravis and paroxysmal nocturnal hemoglobinuria, along with a Phase Ib trial for patients with renal impairment. Additionally, Ra Pharmaceuticals is advancing pre-clinical programs targeting Factor D inhibition for C3 glomerulonephritis and other complement factors for various diseases. The company has established a collaboration with Merck & Co., Inc. to explore orally available cyclic peptides for non-complement cardiovascular targets. Founded in 2008, Ra Pharmaceuticals operates as a subsidiary of UCB SA.

KalVista Pharmaceuticals is a clinical-stage pharmaceutical company based in Cambridge, Massachusetts, focused on discovering, developing, and commercializing small molecule protease inhibitors for diseases with unmet medical needs. The company's primary product portfolio features plasma kallikrein inhibitors aimed at treating hereditary angioedema (HAE) and diabetic macular edema (DME), a major cause of visual loss in adults. KalVista's notable products include KVD001, a plasma kallikrein inhibitor that has completed a Phase II clinical trial for DME, and KVD900, currently in Phase II trials for HAE attacks. Additionally, KVD824, an oral plasma kallikrein inhibitor, has completed a Phase I trial for both DME and HAE. The company also has a preclinical pipeline that includes a novel oral Factor XIIa inhibitor program, enhancing its commitment to addressing diverse therapeutic challenges.

Collegium Pharmaceutical is a specialty pharmaceutical company that develops and commercializes products aimed at addressing pain management while mitigating the risks of prescription drug abuse. The company utilizes its proprietary DETERx platform technology, which ensures that its formulations maintain extended-release and safety profiles even when subjected to common methods of misuse such as chewing or crushing. Collegium's product lineup includes Xtampza ER, an abuse-deterrent, extended-release oral formulation of oxycodone, and Nucynta products, which consist of both extended-release and immediate-release formulations of tapentadol. These offerings are designed to manage chronic pain and provide effective treatment solutions while incorporating tamper-resistant features to combat non-medical use. Headquartered in Stoughton, Massachusetts, Collegium Pharmaceutical was incorporated in 2002 and has focused on building a portfolio of innovative products that align with evolving patient needs and regulatory standards.

Agile Therapeutics is a women's healthcare company dedicated to addressing the unmet health needs of women through the development and commercialization of prescription contraceptive products. The company's lead product, Twirla, is a once-weekly transdermal patch that delivers a combination of levonorgestrel and ethinylestradiol, providing a non-daily contraceptive option. In addition to Twirla, Agile Therapeutics is advancing a pipeline of related products, including various regimens designed to offer customizable menstrual cycle management options, such as AG200-ER for extended cycles and AG200-SP for lighter periods. The firm's proprietary Skinfusion technology facilitates effective drug delivery through the skin. Headquartered in Princeton, New Jersey, Agile Therapeutics aims to enhance women's reproductive health by providing innovative contraceptive solutions.

Strongbridge Biopharma plc is a commercial-stage biopharmaceutical company dedicated to developing and commercializing therapies for rare diseases with significant unmet medical needs. The company markets Keveyis, an oral carbonic anhydrase inhibitor used to treat various forms of primary periodic paralysis in the United States. Its clinical pipeline includes Recorlev, a cortisol synthesis inhibitor currently in Phase III trials for endogenous Cushing's syndrome, and Veldoreotide, a somatostatin analog that has completed Phase II trials for acromegaly. Founded in 1996 and based in Trevose, Pennsylvania, Strongbridge Biopharma aims to build its portfolio around rare endocrine disorders and plans to utilize a focused sales strategy to reach its target markets. The company is also actively seeking to in-license or acquire complementary products to enhance its rare disease franchise.

Moderna, Inc. is a biotechnology company based in Cambridge, Massachusetts, that specializes in developing therapeutics and vaccines using messenger RNA technology. Founded in 2010, the company focuses on various therapeutic areas, including infectious diseases, oncology, rare genetic disorders, and cardiovascular diseases. As of September 2024, Moderna has 40 mRNA development candidates in clinical development, demonstrating its commitment to advancing medical science. The company's mRNA platform has gained significant recognition, particularly with the authorization of its COVID-19 vaccine in December 2020. Moderna has established strategic collaborations with notable organizations such as AstraZeneca, Merck & Co., and the Bill & Melinda Gates Foundation. Additionally, it collaborates with Lonza Ltd. and Catalent for the manufacturing of its COVID-19 vaccine. Through its innovative approach, Moderna aims to provide effective solutions for a range of health challenges.

Blueprint Medicines is a biopharmaceutical company dedicated to developing targeted therapies for cancers and rare diseases driven by abnormal kinase activation. Founded in 2008 and based in Cambridge, Massachusetts, the company utilizes its proprietary Insights-to-Validation Platform and chemical library to create small molecule kinase inhibitors that address specific genomic drivers. Key drug candidates include avapritinib for systemic mastocytosis and gastrointestinal stromal tumors, BLU-263 for indolent systemic mastocytosis, and pralsetinib for RET-altered cancers such as non-small cell lung cancer and medullary thyroid carcinoma. Additionally, fisogatinib is under investigation for advanced hepatocellular carcinoma, while BLU-782 targets fibrodysplasia ossificans progressiva. Blueprint Medicines collaborates with several pharmaceutical companies to enhance its research and development efforts.

EyePoint Pharmaceuticals is a specialty biopharmaceutical company focused on developing and commercializing ophthalmic products for the treatment of eye diseases, primarily in the United States and Europe. The company offers several FDA-approved products, including ILUVIEN for diabetic macular edema, YUTIQ for chronic non-infectious uveitis, DEXYCU for post-operative ocular inflammation, and Retisert for posterior segment uveitis. EyePoint is also advancing its pipeline with EYP-1901, a bioerodible implant targeting wet age-related macular degeneration and related conditions, as well as YUTIQ50 for uveitis. The company utilizes its proprietary Durasert and Verisome technology platforms to create sustained-release drug delivery systems. EyePoint has established strategic collaborations with various organizations and engages in a commercial alliance for the promotion of its products. Founded in 1987 and headquartered in Watertown, Massachusetts, EyePoint was previously known as pSivida Corp.

Larimar Therapeutics, Inc. is a clinical-stage biotechnology company based in Bala Cynwyd, Pennsylvania, dedicated to developing treatments for complex rare diseases. Its primary focus is on Friedreich's ataxia, a rare and progressive genetic disorder, for which it is advancing its lead compound, CTI-1601, through a Phase 1 clinical program. CTI-1601 is designed as a recombinant fusion protein that delivers human frataxin, an essential protein, directly to the mitochondria using a novel cell-penetrating peptide technology. In addition to Friedreich's ataxia, Larimar aims to leverage its intracellular delivery platform to create additional fusion proteins targeting other rare diseases associated with deficiencies in bioactive compounds.

Dicerna Pharmaceuticals is a biotechnology company dedicated to the discovery and development of innovative treatments for rare inherited diseases and other conditions affecting the liver. Utilizing its proprietary ribonucleic acid interference (RNAi) technology, specifically the GalXC platform, Dicerna focuses on silencing disease-causing genes to develop pharmaceuticals for various therapeutic areas, including liver diseases, cardiovascular diseases, and cancers. Key product candidates include nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B virus infection, and additional programs targeting undisclosed rare liver diseases. The company has established strategic collaborations with several major pharmaceutical firms to enhance its research and development efforts. Founded in 2006 and headquartered in Lexington, Massachusetts, Dicerna Pharmaceuticals aims to address significant unmet medical needs through its innovative approaches.

Bluebird Bio is a biotechnology company focused on the research, development, and commercialization of innovative gene therapies for severe genetic diseases and cancer. Its gene therapy programs include LentiGlobin, aimed at treating β-thalassemia and sickle cell disease, and Lenti-D, for cerebral adrenoleukodystrophy. In oncology, Bluebird Bio is developing chimeric antigen receptor T (CAR T) cell therapies, specifically bb2121 and bb21217, for multiple myeloma. The company collaborates with several partners, including Bristol-Myers Squibb and Regeneron Pharmaceuticals, to discover and commercialize disease-altering therapies. Additional collaborations with organizations such as Medigene AG and Gritstone Oncology focus on developing T cell receptor product candidates for cancer treatment. Founded in 1992 and headquartered in Cambridge, Massachusetts, Bluebird Bio aims to fundamentally address the genetic basis of diseases through its proprietary lentiviral vector gene addition platform. Revenue is generated from collaboration agreements, research fees, license fees, and grants.

OvaScience, Inc. was a fertility company focused on developing innovative treatments for women facing infertility challenges. The company was founded in 2011 and was headquartered in Waltham, Massachusetts. OvaScience specialized in proprietary technology derived from the discovery of egg precursor (EggPC) cells, which allowed for the enhancement of egg quality and improved outcomes in in vitro fertilization (IVF). Their treatment options included OvaPrime, which aimed to restore a woman's egg production using her own EggPC cells, OvaTure, designed to mature these cells into fertilizable eggs outside the body, and AUGMENT, which utilized mitochondria from EggPC cells to enhance fertilization and pregnancy rates. The company also explored OvaXon for preventing inherited diseases. OvaScience was co-founded by a team of experts from Harvard Medical School and was backed by prominent life science investors. In December 2018, OvaScience was acquired by Millendo Therapeutics in a reverse merger.

Seres Therapeutics is a clinical-stage biotherapeutic company based in Cambridge, Massachusetts, that specializes in developing ecobiotic microbiome therapeutics aimed at restoring health by correcting dysbiosis in the microbiome. The company is advancing several product candidates, with its lead therapy, SER-109, currently in Phase III clinical trials to prevent recurrence of Clostridium difficile infection (CDI). Additional candidates in development include SER-287 for ulcerative colitis, SER-401 for use with checkpoint inhibitors in metastatic melanoma, and SER-301 for inflammatory bowel disease. The company is also working on SER-262 to address initial CDI recurrences and SER-155 to support patients after allogeneic hematopoietic stem cell transplants. Seres Therapeutics has established collaboration agreements with organizations such as Nestec Ltd. and Memorial Sloan Kettering Cancer Center, as well as partnerships with AstraZeneca. Founded in 2010, the company has evolved from its original name, Seres Health, Inc., reflecting its focus on microbiome-based therapeutic solutions.