RA Capital Management

Jade Biosciences is a biopharmaceutical company dedicated to developing innovative therapies for unmet needs in autoimmune diseases. Its lead asset, JADE-001, targets the APRIL pathway for treating IgA nephropathy, with initial clinical trials planned. Additionally, Jade has two preclinical antibody discovery programs, JADE-002 and JADE-003. The company was founded upon assets licensed from Paragon Therapeutics.

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.

Vivace Therapeutics, Inc. is a drug discovery and development company focused on creating cancer therapies that target the Hippo pathway, which plays a crucial role in tissue regeneration and organ development. Established in 2014 and located in the San Francisco Bay Area, Vivace Therapeutics employs an innovative approach to develop small molecule treatments aimed at improving patient outcomes in oncology. The company collaborates with leading academic institutions to advance research and develop therapies that can be used alone or in combination with existing treatments. With a commitment to enhancing cancer care, Vivace Therapeutics is dedicated to delivering novel solutions that can help patients combat cancer more effectively.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Bambusa Therapeutics operates as biotechnology company, that focuses on developing bispecific antibodies.

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.

Verdiva Bio is a biopharmaceutical company specializing in the development of innovative therapies for patients with obesity and cardiometabolic disorders. The company focuses on creating more patient-friendly treatment options, with a particular emphasis on gut-brain biology and amylin molecules, aiming to improve efficacy, tolerability, and weight loss 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.

Cidara Therapeutics is a biotechnology company based in San Diego, California, established in 2012. The company specializes in the discovery and development of innovative anti-infective therapies for serious diseases, particularly those that affect individuals with compromised immune systems. Its lead candidate, rezafungin acetate, is an antifungal agent designed to treat and prevent severe invasive fungal infections, such as candidemia and invasive candidiasis, which carry significant mortality risks. In addition to its antifungal efforts, Cidara is advancing its proprietary Cloudbreak platform, which focuses on developing antiviral Fc-conjugates aimed at preventing and treating various viral infections, including influenza, RSV, HIV, and coronavirus. Cidara's mission is to transform treatment and prevention strategies for life-threatening illnesses.

Trace Neuroscience is a biopharmaceutical company dedicated to advancing genetic medicine for patients with neurodegenerative diseases. It specializes in developing targeted antisense oligonucleotide therapies, with its lead product aimed at restoring UNC13A protein function in individuals with Amyotrophic Lateral Sclerosis (ALS) caused by TDP-43 mislocalization. This therapy corrects UNC13A mRNA splicing, potentially improving muscle function and extending survival for these patients.

Alentis Therapeutics AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, focused on developing innovative therapies for advanced liver diseases, including liver fibrosis, cirrhosis, and liver cancer. Established in 2019, Alentis is recognized for its pioneering work in targeting CLDN1+ cancers through the use of anti-CLDN1 antibody-drug conjugates and other therapeutic modalities. The company's research platform incorporates clinically relevant read-outs and single-cell RNA sequencing of patient liver tissues, which aids in understanding and treating fibrotic diseases. Alentis aims to modify and reverse disease progression, ultimately addressing significant health challenges associated with liver-related 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.

Superluminal Medicines is a biotechnology company specializing in generative biology and chemistry. It employs a unique platform that combines deep biological and chemical expertise, machine learning, and proprietary big data infrastructure to rapidly generate candidate-ready compounds for drug discovery and development. By integrating high-throughput experimental data with advanced algorithms, the company aims to accelerate the identification and optimization of novel drug candidates, improving efficiency and success rates in small molecule drug development compared to traditional methods.

Outpace Bio is a biotechnology company that creates smart cell therapies aimed at improving efficacy and safety. It offers a platform that combines custom-designed biological functions with cellular control modalities that program the right response at the right time and place. It creates a mechanism-driven solution tailor-made to improve efficacy and safety. The company collaborates with Lyell Immunopharma to develop and commercialize a potential immune cell therapy for the treatment of cancer, auto-immune diseases, infectious diseases, degenerative diseases, regenerative medicine, metabolic disorders, and genetic disorders, enabling healthcare providers to cure a wide range of diseases as per patient's need.

CellCentric Ltd. is a biotechnology company based in Cambridge, United Kingdom, focused on developing innovative cancer therapeutic products. Established in 2003, the company specializes in small-molecule inhibitors that target p300 and CBP proteins, as well as a deubiquitinase enzyme closely linked to prostate cancer. This enzyme modulates the androgen receptor pathway, which is critical in addressing resistance mechanisms encountered with existing prostate cancer treatments. CellCentric's therapies aim to treat various cancers, including castrate-resistant prostate cancer, lung cancer, bladder cancer, non-small cell lung cancer, breast cancer, and colon cancer. By targeting specific pathways and mechanisms, CellCentric seeks to provide effective treatment options for patients with challenging cancer types.

Gameto is a biotechnology company focused on improving female reproductive health through innovative therapies. Recognizing that women's health is an underserved area with limited treatment options, Gameto aims to develop advanced solutions that enhance patient outcomes. The company utilizes cellular engineering to create female reproductive cell lines, employing proprietary combinations of transcription factors to reprogram human induced pluripotent stem cells (hiPSCs) into engineered ovarian and endometrial cell lines that function similarly to natural cells. This platform supports a portfolio of cellular therapeutics targeting various female reproductive diseases. Gameto's flagship program, Fertilo, is designed to enhance the processes of in vitro fertilization (IVF) and egg freezing, making them shorter, safer, and more effective. Additional applications of Gameto's technology include Deovo, an organoid of the female reproductive system, and Ameno, a cell-based therapeutic aimed at alleviating the health issues associated with primary ovarian insufficiency and menopause.

Bicycle Therapeutics is a clinical-stage biopharmaceutical company focused on developing a novel class of medicines known as Bicycles, which are synthetic short peptides designed to stabilize their structural geometry through two loops. The company primarily targets oncology indications with significant unmet medical needs. Its lead product candidate, BT1718, is a Bicycle Toxin Conjugate currently undergoing Phase I/IIa clinical trials aimed at tumors expressing Membrane Type 1 matrix metalloprotease. Other oncology candidates include BT5528, also in Phase I/IIa trials targeting EphA2, and BT8009, which is in preclinical studies for Nectin-4. Additionally, Bicycle Therapeutics is developing THR-149, a plasma kallikrein inhibitor that has completed Phase I trials for diabetic macular edema. The company collaborates with various biopharmaceutical organizations to advance programs in areas like anti-infectives and cardiovascular health. Founded in 2009, Bicycle Therapeutics is headquartered in Cambridge, United Kingdom.

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.

AltruBio is a biopharmaceutical company focused on developing targeted antibody therapeutics for cancer and immune-related inflammatory diseases. Established in Delaware with its principal office in the San Francisco Bay Area, AltruBio integrates global resources to advance its research and development initiatives. The company has its roots in Taipei, Taiwan, where it has been working since June 2000 on discovering and developing novel therapeutics to address unmet medical needs. AltruBio employs a sophisticated discovery platform to identify and validate new antibody therapeutics. Currently, the company is developing several potential therapeutic antibodies that target a range of conditions, including autoimmune diseases and various types of cancer, such as pancreatic, stomach, colorectal, ovarian, and lung cancers, at different stages of development.

Medicenna Therapeutics Corp. is a clinical-stage immunotherapy company based in Toronto, Canada, focused on the development and commercialization of novel cytokine-based therapies for cancer treatment. The company specializes in Empowered Cytokines (EC) and Superkines, including its lead product, MDNA55, which is currently undergoing Phase 2b clinical trials for recurrent glioblastoma. Medicenna's pipeline also features MDNA57, targeting solid tumors; MDNA109, an IL-2 agonist for cancer immunotherapy; MDNA209, an IL-2 antagonist for autoimmune diseases; MDNA413, a dual IL-4/IL-13 antagonist for various solid tumors and inflammatory conditions; and MDNA132, an IL-13 agonist aimed at treating solid tumors. Its innovative technology platform allows for precise delivery of therapeutic agents while minimizing damage to healthy cells, positioning Medicenna as a significant player in the immuno-oncology space. The company was founded in 2012 and continues to expand its research and development efforts to address a wide range of cancers and related diseases.

Zura Bio is a clinical-stage biotechnology company that develops innovative therapies for immune and inflammatory disorders. The company focuses on advancing its programs through phase 2 development, specifically targeting auto-immune illnesses. One of its key assets, ZB-168, is an anti-IL7R inhibitor designed to address disorders linked to the biological pathways of IL7 and TSLP, building on data from phase 1b studies related to type 1 diabetes. Additionally, Zura Bio is progressing the phase 2 clinical development of torudokimab, a human-affinity monoclonal antibody that neutralizes IL33. Through these efforts, Zura Bio aims to create effective therapeutic solutions for patients suffering from various immune-related conditions.

TORL BioTherapeutics is a clinical-stage biopharmaceutical company dedicated to developing innovative antibody-based medicines aimed at improving outcomes for cancer patients. The company specializes in creating antibody-drug conjugates (ADCs) and monoclonal antibodies (mAbs) to address oncologic diseases that exhibit significant unmet medical needs. TORL BioTherapeutics employs a strategic approach that includes target identification and early discovery work, allowing them to license promising drug programs and develop proprietary therapies with optimized characteristics. Through this pipeline, TORL BioTherapeutics is positioned to advance novel treatments that could significantly enhance patient care in oncology.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Avalo Therapeutics is a clinical-stage biopharmaceutical company that focuses on developing highly targeted therapeutics for immune dysregulation and other areas of significant unmet clinical need. The company employs a precision medicine approach in its research and development efforts. Its lead asset, AVTX-009, is an anti-IL-1B monoclonal antibody designed to address inflammatory diseases. In addition to AVTX-009, Avalo's pipeline includes quisovalimab, an anti-LIGHT monoclonal antibody, and AVTX-008, a BTLA agonist fusion protein, which further exemplify its commitment to advancing innovative treatments for complex medical conditions.

Cybin is a biopharmaceutical company dedicated to advancing research and development in psychedelic and medicinal mushrooms. It focuses on creating safe and effective therapeutics for mental health issues, with an emphasis on psilocybin-based products. The company is actively launching these products in jurisdictions where they are permitted and is engaged in clinical studies across North America and other regions. Through strategic partnerships with academic and institutional entities, Cybin seeks to develop novel compounds and delivery mechanisms, aiming to provide innovative treatments for various psychiatric and neurological conditions.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to address aging and age-related diseases. The company utilizes a systems biology and artificial intelligence platform to identify key drug targets that influence aging and analyze extensive datasets to uncover the molecular drivers of age-related conditions. BioAge Labs is advancing several drug candidates, including BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor designed to activate genes involved in critical biological processes such as tissue regeneration and vascular remodeling. Additionally, BGE-175, an orally administered inhibitor of the prostaglandin D2 DP1 signaling pathway, aims to mitigate risks associated with immune aging and respiratory infections. Founded in 2015 and based in Richmond, California, BioAge Labs is committed to discovering and developing innovative treatments that harness the biology of human aging.

Tenaya Therapeutics is a biotechnology company based in South San Francisco, California, focused on developing innovative therapies for heart disease, particularly heart failure. Established in 2016, the company employs three main platforms to address the underlying causes of heart disease: a cellular regeneration platform that reprograms cardiac fibroblasts into cardiomyocytes using proprietary transcription factors, a gene therapy platform for targeted delivery of therapeutic payloads, and a precision medicine approach tailored to individual patients. Through these platforms, Tenaya Therapeutics aims to enable the regeneration of heart tissue and tackle various forms of cardiomyopathies, leveraging advanced research in cardiac development and regeneration.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Nature’s Toolbox is a biotechnology company based in New Mexico that has developed a proprietary host-independent biological manufacturing and development platform. Founded in 2015, the company focuses on identifying, evaluating, and prioritizing naturally occurring drug discovery targets. Its platform provides high-throughput screening capabilities for the rapid prototyping of biological materials, allowing for real-time metabolic profiling of bacteria, fungi, and plants. This technology facilitates the discovery, characterization, and production of bioactive natural products, which can be applied to a range of applications, including protein-based therapies for cancer and autoimmune disorders. By enhancing reproducibility and reducing the resource footprint, Nature’s Toolbox aims to accelerate the drug development process for its clients.

Aiolos Bio is a biotechnology company specializing in the development of therapies for respiratory diseases and immune conditions. Its primary focus is on advancing AIO-001, a monoclonal antibody therapy for moderate-to-severe asthma, designed for dosing every 6 months. The company, founded by industry veterans, aims to address unmet treatment needs and improve patient outcomes by providing more efficacious and less burdensome therapies.

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.

Hyku is a biotechnology company focused on developing small molecule therapeutics that target disease-causing proteins through precise covalent binding. Utilizing its innovative platform, Hyku identifies molecules that covalently bind to non-cysteine amino acids, facilitating the creation of novel therapies. These therapies aim to address significant challenges in drug development, including issues related to selectivity, druggability, and resistance. By pushing the boundaries of covalent medicine, Hyku seeks to create effective treatments that can improve patient outcomes and advance the field of therapeutic development.

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.

Star Therapeutics is a biotechnology company focused on developing innovative therapies for a wide range of rare diseases. The company aims to identify diseases that share common biological characteristics and create novel therapeutics that can effectively treat multiple conditions with a single treatment. This approach not only addresses the needs of patients suffering from rare diseases but also presents a unique opportunity for pharmaceutical companies to broaden their therapeutic offerings within a streamlined pipeline. By concentrating on life-changing therapies, Star Therapeutics aspires to make a significant impact on the lives of millions of patients.

Superluminal Medicines is a biotechnology company specializing in generative biology and chemistry. It employs a unique platform that combines deep biological and chemical expertise, machine learning, and proprietary big data infrastructure to rapidly generate candidate-ready compounds for drug discovery and development. By integrating high-throughput experimental data with advanced algorithms, the company aims to accelerate the identification and optimization of novel drug candidates, improving efficiency and success rates in small molecule drug development compared to traditional methods.

ExcepGen Ltd is a biotechnology company focused on developing full-length human proteins for drug discovery. Founded in 2017 and based in London, the company employs engineered human cell lines to produce these proteins, facilitating research and development in therapeutics. By utilizing proprietary technology, ExcepGen aims to enhance the effectiveness of mRNA therapeutics, optimizing cellular responses to improve therapeutic outcomes. This approach allows laboratories to minimize development time, accelerate high-throughput screening processes, and make informed decisions regarding their therapeutic pipelines. With a commitment to addressing fundamental challenges in drug discovery, ExcepGen is positioned to contribute significantly to the advancement of innovative treatments.

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.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company based in Shanghai, China, focused on discovering and developing cell therapies for cancer treatment. The company’s lead product candidates include GC012F, a dual-target autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia and is also being investigated for relapsed or refractory B cell non-Hodgkin’s lymphoma. Additionally, Gracell is developing GC007F for B cell non-Hodgkin's lymphoma and GC027, an allogeneic CAR-T product candidate for adult T cell acute lymphoblastic leukemia. The company also has GC007g, a donor-derived therapy for relapsed or refractory B cell acute lymphoblastic leukemia, and a pipeline of earlier stage candidates targeting other cancer types, including ovarian cancer and breast cancer. Founded in 2017, Gracell aims to provide innovative cellular therapeutics that enhance treatment options for patients suffering from various hematological malignancies and solid tumors.

Septerna is a biotechnology company focused on discovering and advancing novel small-molecule medicines that target G protein-coupled receptors (GPCRs). The company aims to improve patient outcomes by integrating innovative drug discovery technologies with GPCR drug targets. Utilizing its Native Complex platform, Septerna facilitates industrial-scale drug discovery through advanced screening technologies and structure-based drug design. This approach enables medical researchers to efficiently identify treatments for a variety of diseases.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.

Pluton Biosciences is a bioscience research company that specializes in the testing and discovery of microbes for the development of various products, primarily focusing on biopesticides and pharmaceuticals. Utilizing an innovative platform, the company mines and clones microbes to generate a range of beneficial products aimed at enhancing biopesticides, crop protection, human health, and bioremediation. By harnessing the potential of bacteria, fungi, and viruses, Pluton Biosciences enables medical researchers and agricultural professionals to explore sustainable and all-natural solutions for various applications, contributing to advancements in both environmental and health-related sectors.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Alentis Therapeutics AG is a clinical-stage biopharmaceutical company based in Basel, Switzerland, focused on developing innovative therapies for advanced liver diseases, including liver fibrosis, cirrhosis, and liver cancer. Established in 2019, Alentis is recognized for its pioneering work in targeting CLDN1+ cancers through the use of anti-CLDN1 antibody-drug conjugates and other therapeutic modalities. The company's research platform incorporates clinically relevant read-outs and single-cell RNA sequencing of patient liver tissues, which aids in understanding and treating fibrotic diseases. Alentis aims to modify and reverse disease progression, ultimately addressing significant health challenges associated with liver-related conditions.

Capacity Bio is a therapeutics company dedicated to advancing the field of mitophagy, which involves the regulation of mitochondrial quality control. The company is developing a comprehensive platform aimed at creating innovative mitophagy therapeutics. By focusing on fundamental biological processes, Capacity Bio seeks to provide radical therapeutic benefits that can restore mitochondrial function in various diseases, thereby addressing critical healthcare challenges.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Unicycive Therapeutics provides development programs that focus on treating kidney diseases with significant unmet medical needs. It aims to open the way for a broad range of disease-modifying therapies by modifying the intracellular and extracellular concentrations of these various electrolytes and ions in Chronic Kidney Disease and Gitelman’s Syndrome and by addressing the underlying mitochondrial pathology and inflammation associated with Acute Kidney Disease. Unicycive Therapeutics was founded in 2016 and is headquartered in Los Altos, California.

TeraPore Technologies, Inc., established in 2013 and headquartered in South San Francisco, specializes in developing advanced membrane technology for bioprocessing applications. Its innovative solution enables rapid and efficient purification of biological fluids, facilitating the separation of high-value products with uniform pores and large pore densities, thereby reducing processing times. The company's technology is designed to address bioseparations challenges, offering targeted and uniform nanofiltration capabilities for consistent and controllable purification processes.

Avilar Therapeutics is a biopharmaceutical company dedicated to the discovery and development of extracellular protein degraders, which represent an innovative approach to targeted protein degradation. The company has created an integrated discovery platform that extends the concept of protein degradation beyond intracellular proteins. Avilar’s novel degraders are designed to bind to disease-causing proteins in circulation and shuttle them to the hepatocyte endolysosome for degradation. This mechanism aims to facilitate the treatment of serious diseases by removing unwanted proteins from the body, thereby offering potential therapeutic benefits in various medical 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.

NewAmsterdam Pharma, established in 2019 and headquartered in Naarden, the Netherlands, is a late-stage biopharmaceutical company dedicated to improving patient care in cardio-metabolic disease populations. The company's primary focus is the development of transformative therapies, with its lead product, obicetrapib, being a next-generation, oral, low-dose CETP inhibitor designed to lower LDL-C levels more effectively than current treatments, particularly for patients with statin intolerance or Arteriosclerotic Cardiovascular Disease/Familial Hypercholesterolemia (ASCVD/FH) on maximally-tolerated statin therapy.

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.

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 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

Rivus Pharmaceuticals is a biopharmaceutical company focused on enhancing cardio-metabolic health through the development of controlled metabolic accelerators (CMAs). These innovative therapeutics aim to improve cellular metabolism and target the root causes of prevalent metabolic and cardiovascular diseases. By providing oral small-molecule treatments, Rivus addresses critical metabolic risk factors associated with cardiovascular mortality and morbidity, thereby contributing to better health outcomes for patients suffering from these conditions.

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.

Carmot Therapeutics, Inc. is a clinical-stage biotechnology company based in Berkeley, California, with an additional office in San Francisco. Founded in 2008, the company focuses on discovering and developing therapies for metabolic diseases, including obesity and diabetes. Carmot employs a proprietary drug discovery approach known as Chemotype Evolution, which enables the exploration of novel chemical and biological frontiers. The company is advancing a portfolio of clinical-stage drug candidates, including CT-388, a once-weekly injectable dual GLP-1/GIP receptor agonist; CT-996, a once-daily oral GLP-1 receptor agonist; and CT-868, a once-daily injectable dual GLP-1/GIP receptor agonist for treating type 1 diabetes in patients with overweight or obesity. These innovative therapies aim to enhance glucose clearance and promote weight loss, addressing the fundamental causes of metabolic diseases.

NAPIGEN is a biotechnology company focused on innovative genome engineering across a variety of organisms, including plants, microbes, and animals. The company specializes in targeting organelles such as mitochondria and chloroplasts, which contain DNA essential for cellular energy production. By harnessing the capabilities of these organelles, NAPIGEN aims to enhance biological functions and contribute to advancements in agriculture, environmental sustainability, and other sectors, ultimately improving the quality of life in society.

Wave Life Sciences Ltd. is a clinical-stage genetic medicine company focused on developing innovative RNA-based therapies. Utilizing its proprietary PRISM platform, the company designs, optimizes, and produces stereopure oligonucleotides aimed at addressing genetic defects. By targeting ribonucleic acid (RNA), Wave aims to either diminish the production of harmful proteins or convert dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly disorders affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to further advance its therapeutic programs. Founded in 2012 and headquartered in Singapore, Wave Life Sciences is dedicated to delivering transformative treatments for patients with genetically defined diseases.

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.

CDR-Life is a Zurich-based biotech company specializing in the development of innovative therapeutic antibody fragments, with particular emphasis on immuno-oncology and ophthalmology. Operating from the Bio-Technopark in Schlieren-Zurich, the company focuses on creating tumor-selective immunotherapies. Its technology is designed to produce MHC-specific antibodies that target tumor-specific intracellular antigens, thereby enabling healthcare providers to deliver more precise and effective treatments for cancer patients.

Triana Biomedicines is focused on discovering and developing molecular glues, a novel therapeutic approach aimed at regulating challenging disease targets. The company employs a sophisticated drug discovery engine that combines high-resolution structural insights, advanced in silico tools, and custom chemical libraries. This platform is designed to stabilize existing protein interactions or create new ones, thereby influencing the functionality of disease targets. Triana has successfully validated its technology and is currently advancing multiple research programs across various disease areas, positioning itself to contribute significantly to therapeutic innovation.

Synthego Corporation is a genome engineering company focused on advancing life science research and therapeutic development to improve human health. Founded in 2012 and headquartered in Redwood City, California, Synthego offers a comprehensive platform that integrates proprietary hardware, software, bioinformatics, chemistries, and molecular biology. The company's product portfolio includes engineered cells, CRISPR kits, and bioinformatics tools designed for various applications such as pathway analysis, target validation, disease modeling, and diagnostics. By leveraging machine learning and automation, Synthego enables researchers in biotechnology and academia to conduct efficient and precise gene editing, facilitating rapid advancements in drug discovery and development. The company serves a global customer base, distributing its products through various channels, including online sales and partnerships in multiple countries.

Star Therapeutics is a biotechnology company focused on developing innovative therapies for a wide range of rare diseases. The company aims to identify diseases that share common biological characteristics and create novel therapeutics that can effectively treat multiple conditions with a single treatment. This approach not only addresses the needs of patients suffering from rare diseases but also presents a unique opportunity for pharmaceutical companies to broaden their therapeutic offerings within a streamlined pipeline. By concentrating on life-changing therapies, Star Therapeutics aspires to make a significant impact on the lives of millions of patients.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Metagenomi, Inc. is a biotechnology company focused on developing gene editing systems aimed at treating genetic diseases. Founded in 2016 and based in Emeryville, California, Metagenomi utilizes metagenomics and machine learning to uncover novel genome editing technologies derived from previously uncharacterized organisms. The company's innovative discovery platform enables the creation of highly efficient and specific gene editing tools, including programmable nucleases, base editors, and advanced integration systems such as prime editing and CRISPR-associated transposases. By harnessing these naturally evolved systems, Metagenomi seeks to address a wide range of genetic mutations that traditional genome engineering approaches have struggled to target, with the ultimate goal of providing transformative therapies for patients globally.

AmbAgon Therapeutics is a biotechnology company focused on developing small-molecule therapeutics for cancer treatment. The company specializes in research aimed at stabilizing the interactions of oncogenic proteins, thereby enhancing the activity of tumor-suppressing proteins. By targeting these proteins, AmbAgon Therapeutics seeks to address significant unmet clinical needs in oncology that conventional treatment methods have struggled to meet. Founded by Christian Ottmann, Lucas Brunsveld, and Michelle Arkin, the company aims to provide innovative solutions in cancer care through its unique approach to drug development.

Nodexus is a biotech startup focused on providing innovative solutions for live single-cell isolation through its NX One platform. This platform features a low-cost, low-infrastructure hardware system paired with single-use disposable microfluidic cartridges, facilitating various applications in the biopharma, clinical research, and academic fields. Nodexus's technology supports critical workflows such as gene editing, cell line and antibody development, and studies of tumor heterogeneity. By offering affordable automated cell isolation, Nodexus aims to enhance biological research and development, making advanced methodologies more accessible to a wider range of clients.

Avilar Therapeutics is a biopharmaceutical company dedicated to the discovery and development of extracellular protein degraders, which represent an innovative approach to targeted protein degradation. The company has created an integrated discovery platform that extends the concept of protein degradation beyond intracellular proteins. Avilar’s novel degraders are designed to bind to disease-causing proteins in circulation and shuttle them to the hepatocyte endolysosome for degradation. This mechanism aims to facilitate the treatment of serious diseases by removing unwanted proteins from the body, thereby offering potential therapeutic benefits in various medical conditions.

Acrivon Therapeutics is a clinical-stage biopharmaceutical company focused on precision oncology, developing therapeutics that target challenging cancer types without straightforward single-gene mutations. The company employs a proteomics-based platform to identify patients whose tumors are likely to respond to specific treatments. Acrivon's pipeline includes ACR-368, also known as prexasertib, which is currently in Phase 2 trials, alongside preclinical programs aimed at key components of DNA damage response and cell cycle regulation, such as the WEE1 and PKMYT1 proteins. Acrivon seeks to expedite the development of both in-licensed clinical-stage assets and its internally developed programs through accelerated registration trials, aiming to enhance treatment success in oncology.

Xenikos is focused on developing innovative immunotherapy treatments aimed at addressing serious immune diseases and complications arising from transplants. The company specializes in anti-T-cell antibodies that can effectively reset the immune system. This therapeutic approach involves injecting a specific agent that targets and eliminates adult T cells, particularly those that are activated, thereby enhancing treatment outcomes for patients. By leveraging this unique mechanism, Xenikos seeks to restore health and improve the quality of life for individuals facing significant immune-related challenges.

DICE Therapeutics is a biopharmaceutical company that utilizes its proprietary technology platform to develop a pipeline of novel oral therapeutic candidates aimed at treating chronic diseases, particularly in the field of immunology. The company focuses on creating oral therapeutics that target well-established proteins, intending to achieve efficacy similar to existing systemic biologics. DICE's DELSCAPE platform is specifically designed to discover selective oral small molecules capable of modulating protein-protein interactions effectively. Its lead candidate, S011806, is an oral antagonist targeting interleukin-17, a key player in various immunological conditions. In addition to S011806, DICE is developing other oral therapeutic candidates that target α4ß7 integrin and αVß1/αVß6 integrin, aimed at treating inflammatory bowel disease and idiopathic pulmonary fibrosis, respectively.

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.

Frontier Medicines Corporation is a pre-clinical stage biopharmaceutical company based in South San Francisco, California, founded in 2018. The company focuses on the discovery and development of medicines targeting cancer-causing proteins through its innovative chemoproteomics platform. This platform utilizes advanced computational methods and machine learning to identify and pharmacologically target new binding pockets on proteins, facilitating the development of small-molecule drugs. By enhancing the accessibility of these proteins, Frontier Medicines aims to provide effective treatments that can redefine the management of debilitating diseases.

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.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

Nautilus Biotechnology is a development-stage life sciences company focused on advancing proteomics, the study of proteins and their functions, to enhance therapeutic development and medical diagnostics. The company has created a platform technology that aims to quantify and unravel the complexities of the human proteome. Its flagship product, the Proteomic Analysis System, is a prototype single-molecule instrument designed to facilitate these efforts. The team at Nautilus comprises experts from various disciplines, including protein chemistry, molecular biology, and data science, enabling a multifaceted approach to overcoming challenges in the field. By harnessing this diverse expertise, Nautilus aims to revolutionize drug development and improve healthcare management through personalized and predictive medicine.

Synthekine Inc. is a biotechnology company focused on developing innovative cytokine therapies and immunotherapies for cancer and autoimmune disorders. Established in 2018 and located in Menlo Park, California, the company employs advanced platforms such as engineered partial agonists and orthogonal cell therapies. Its Synthekine Platform utilizes surrogate agonists instead of traditional mutant cytokines, allowing for the creation of therapeutics that enhance efficacy while minimizing side effects. The product pipeline includes STK-009, an orthogonal ligand, and SYNCAR-001, a CAR-T therapy targeting CD-19, along with STK-012, which consists of partial agonists of IL-2. Synthekine aims to revolutionize treatment paradigms by harnessing the principles of cytokine partial agonism and immunological specificity, leading to novel immunotherapies that improve patient outcomes.

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.

Affinia Therapeutics Inc. is focused on developing gene therapies for various diseases, particularly those affecting the muscles and central nervous system. The company specializes in creating adeno-associated virus (AAV) vectors, leveraging advanced techniques in synthetic and systems biology, as well as high-throughput screening and tissue and single-cell resolution. Founded in 2019 and based in Waltham, Massachusetts, Affinia aims to address significant unmet medical needs by providing rationally designed gene therapies with improved properties. The company's innovative approach seeks to offer effective treatments for devastating conditions that currently lack adequate therapeutic options.

Boundless Bio is a clinical-stage biopharmaceutical company focused on developing innovative therapies for the treatment of aggressive cancers linked to extrachromosomal DNA (ecDNA). Founded in 2018 and based in La Jolla, California, the company aims to address the significant unmet medical needs of patients with oncogene amplified tumors, which affect over 14% of cancer patients. By targeting ecDNA, a key factor in oncogene amplification, Boundless Bio seeks to provide transformative treatments for previously difficult-to-treat cancers. One of its leading candidates, BBI-355, is an oral, selective inhibitor of checkpoint kinase 1 (CHK1), designed to manage the replication and transcription of ecDNA in cancer cells.

Invivyd is a biopharmaceutical company committed to developing antibody-based therapies aimed at providing protection against serious viral infectious diseases. The company focuses on creating solutions that go beyond naturally occurring immunity, particularly in response to viral threats with pandemic potential, starting with SARS-CoV-2. Its product pipeline includes ADG20, which is designed for the treatment and prevention of COVID-19, and VYD222, a monoclonal antibody engineered for enhanced potency and broad coverage. By prioritizing rapid and enduring therapeutic options, Invivyd aims to safeguard vulnerable populations from the severe impacts of circulating viral infections.

Ventus Therapeutics is a biopharmaceutical company focused on discovering and developing novel small molecule medicines aimed at treating autoimmune diseases, inflammatory diseases, and cancer by targeting the innate immune system. The company employs a structural immunology platform that provides deep insights into the mechanisms and molecular structures involved in immune responses. This platform integrates proprietary protein engineering capabilities with advanced rational and structure-based drug design tools, allowing for precise targeting of the innate immune system. Ventus is actively building a pipeline of drug programs that address critical targets within this system, particularly proteins involved in inflammasome and nucleic acid-sensing signaling pathways, thereby enabling the treatment of both acute and chronic inflammatory and immune-related conditions.

Icosavax, Inc. is a biotechnology company based in Seattle, Washington, founded in 2017. The company specializes in developing vaccines using its innovative virus-like particle (VLP) technology platform, which allows for the multivalent display of complex viral antigens. This technology aims to provide broad and durable protection against various infectious diseases, with an initial focus on life-threatening respiratory illnesses. Icosavax's pipeline includes vaccine candidates targeting respiratory syncytial virus (RSV), human metapneumovirus (hMPV), and SARS-CoV-2. The company is dedicated to advancing its VLP technology to discover, develop, and ultimately commercialize effective vaccines against these infectious diseases.

New Equilibrium Biosciences is focused on developing therapeutic drugs aimed at treating cancer and neurodegenerative diseases. The company employs a unique approach that integrates artificial intelligence with quantum chemistry to enhance biophysical experiments. This innovative platform specializes in the research and development of therapeutics that target intrinsically disordered proteins (IDPs), which are often implicated in various neurodegenerative disorders. By utilizing IDP-specific compound libraries, New Equilibrium Biosciences seeks to identify and exploit transient binding pockets on these proteins, facilitating the discovery of lead drug candidates. This approach aims to improve medical procedures and ultimately contribute to advancements in healthcare.

Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics based on CRISPR technology to address genetic disorders. Founded in 2017 and headquartered in Berkeley, California, the company utilizes its proprietary X-Editing (XE) platform, which features highly engineered CRISPR enzymes designed to improve the efficacy, specificity, and deliverability of genome editing. Scribe Therapeutics aims to overcome existing limitations in genetic medicine by creating custom-engineered enzymes and advanced delivery systems. Their approach seeks to establish CRISPR-based therapies as a new standard in clinical care, potentially transforming the treatment landscape for various conditions, including immuno-oncology, degenerative disorders, and enzyme replacement therapy. By prioritizing therapeutic precision and allele-specific targeting, Scribe Therapeutics is committed to expanding access to groundbreaking treatments that can significantly impact patient lives.

Pyxis Oncology, established in 2019 in Boston, Massachusetts, is a biotechnology company specializing in the development of antibody therapeutics for cancer treatment. The company focuses on promoting the body's immune response to cancer by analyzing tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and tumor cell signaling pathways within cold tumors. Pyxis aims to create novel antibody-based immunotherapies to directly kill tumor cells and address underlying pathologies that enable cancer proliferation and immune evasion, with the goal of improving patient outcomes for difficult-to-treat cancers.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

Tenaya Therapeutics is a biotechnology company based in South San Francisco, California, focused on developing innovative therapies for heart disease, particularly heart failure. Established in 2016, the company employs three main platforms to address the underlying causes of heart disease: a cellular regeneration platform that reprograms cardiac fibroblasts into cardiomyocytes using proprietary transcription factors, a gene therapy platform for targeted delivery of therapeutic payloads, and a precision medicine approach tailored to individual patients. Through these platforms, Tenaya Therapeutics aims to enable the regeneration of heart tissue and tackle various forms of cardiomyopathies, leveraging advanced research in cardiac development and regeneration.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Vividion Therapeutics, Inc. is a biotechnology company based in San Diego, California, that focuses on developing innovative therapeutics to address significant unmet medical needs. Established in 2013, Vividion utilizes a pioneering platform for proteome-wide small molecule drug discovery, which combines advanced synthetic chemistry techniques with chemical proteomics. This approach allows the company to expand the druggable proteome and target challenging proteins, ultimately enabling the discovery of transformative treatments for serious illnesses. Vividion's scientific foundation stems from experts in chemical biology and synthetic chemistry at The Scripps Research Institute, reflecting its commitment to advancing therapeutics that can significantly improve patient outcomes.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.