New Enterprise Associates

Abcuro, Inc. is a biotechnology company focused on developing immunotherapies for the treatment of autoimmunity and cancer. Established in 2015 and headquartered in Newton, Massachusetts, Abcuro specializes in creating therapeutic antibodies that target novel pathways in these diseases. The company employs advanced bioinformatics to analyze transcription data from human diseases, enabling them to identify new therapeutic targets within the immune system. By leveraging clinical insights and systematic target validation, Abcuro aims to develop immune-modulatory biotherapeutics that provide innovative treatment options for patients suffering from autoimmune disorders and cancer.

Tune Therapeutics is a biotechnology company focused on developing innovative cell and gene therapies. It employs an epigenetic programming platform to create novel therapeutic pathways, aiming to treat severe diseases without modifying the underlying DNA sequence.

Ouroboros Medicines is a biotechnology company dedicated to immunology, focusing on treating chronic immune-mediated illnesses. It specializes in developing immune reset treatments, using a therapeutic platform that employs T cell engager antibodies to target and deplete specific pathogenic cells. This approach aims to provide extended remissions for patients, without the need for ongoing immunosuppression. The company's strength lies in its integration of scientific knowledge and proven track record in progressing cell-depleting treatments through clinical trials, supported by a robust pipeline.

Orbis Medicines is a biotechnology company focused on advancing drug discovery through its innovative macrocyclic chemistry and computational platform. The company specializes in high-throughput drug discovery, aiming to develop new therapeutics that can significantly improve patient outcomes. By leveraging its unique chemistry and advanced computational techniques, Orbis Medicines seeks to address challenging targets in areas with high unmet medical needs, thereby accelerating the process of bringing effective treatments to market.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, a targeted cancer therapy using gene-engineered allogeneic CAR-NK cells designed to eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A for treating hepatocellular carcinoma.

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

Pathos is a company that focuses on enhancing the drug discovery process through its AI-enabled platform. This self-learning technology leverages big data and artificial intelligence to streamline the identification and validation of drug targets, ultimately establishing their clinical efficacy. By facilitating the exploration of novel concepts, Pathos aims to support drug developers in advancing innovative therapies. The platform is designed to manage the entire end-to-end process of designing and developing new therapeutics, thereby significantly reducing the time required for medication development.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

MBX Biosciences, Inc. is a biotechnology company founded in 2018 and based in Carmel, Indiana. The company specializes in developing therapeutics for rare endocrine disorders, focusing on conditions that currently lack effective treatments. MBX Biosciences aims to discover, develop, and commercialize first-in-class therapies, particularly peptides designed to address endocrine genetic diseases that affect glandular hormone production and the body’s chemical messaging systems.

Cardurion Pharmaceuticals, LLC, established in 2017 and headquartered in Boston, Massachusetts, is a biotechnology company dedicated to developing innovative therapies for heart failure and other cardiovascular diseases. The company is advancing clinical programs that target PDE9 and CaMKII inhibition, utilizing its expertise in cardiovascular signaling pathways to address significant unmet patient needs.

Amber Therapeutics is a biotechnology company that specializes in developing advanced bioelectrical therapies aimed at treating functional disorders of the peripheral nervous system. The company creates a bioelectronic platform that delivers closed-loop bioelectrical treatments, which can sense and adapt to the patient's needs while also providing stimulation. By focusing on conditions such as mixed urinary incontinence, Amber Therapeutics seeks to enhance clinical efficacy and patient outcomes through innovative approaches that emphasize the peripheral nervous system rather than the central nervous system. Its technology has the potential to significantly improve treatment methods and overall patient care in targeted disease areas.

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

Xaira Therapeutics is a biotechnology company based in the San Francisco Bay Area that focuses on leveraging artificial intelligence to enhance drug discovery and development. The company integrates machine learning research, extensive biological and clinical data generation, and therapeutic product development to innovate how diseases are treated. By employing advanced AI methodologies, Xaira aims to transform the traditional drug development process, enabling the creation of more effective therapies. Through its multidisciplinary approach, the company seeks to advance understanding of biology and improve the overall efficiency of developing new treatments.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

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

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Comanche Biopharma is a biopharmaceutical company focused on developing an investigational siRNA medicine aimed at addressing preterm preeclampsia, a serious pregnancy complication. The company is committed to creating safe and effective treatment options that are both evidence-based and affordable, ensuring that all women and their infants can access necessary therapies for life-threatening conditions during pregnancy. By targeting the root causes of preeclampsia, Comanche Biopharma aspires to provide sustainable solutions that improve maternal and fetal health outcomes.

Nexo Therapeutics is a small molecule oncology company focused on discovering and developing innovative drugs for cancer patients who have limited therapeutic options. The company utilizes a unique platform that integrates covalent ligand discovery and chemical biology, allowing it to advance small-molecule therapies aimed at challenging and previously intractable cancer targets. By unlocking a pipeline of novel oncology treatments, Nexo Therapeutics seeks to provide medical professionals with new and effective options to improve patient outcomes in the fight against cancer.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

Tagworks Pharmaceuticals is a privately held biotech company based in the Netherlands, established in 2011 as a spin-out from Philips Healthcare. The company focuses on developing innovative in vivo chemistry to enhance the effectiveness of cancer therapies and to facilitate novel companion diagnostics. Operating from the Radboud University Medical Center in Nijmegen, Tagworks specializes in antibody-based imaging and therapy technologies. Its proprietary approach allows for the selective chemical manipulation of tagged antibodies in vivo, which improves the efficacy of established methods like radioimmuno-imaging and antibody-drug conjugates. This technology enables medical professionals to conduct imaging with significantly reduced radiation exposure for patients.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Launchpad Therapeutics is a precision oncology company focused on developing innovative antibody-based treatments for cancer. The company specializes in creating mutant-selective antibodies designed to target specific mutations found in cancer cells. By leveraging artificial intelligence-driven programs, Launchpad Therapeutics aims to deliver novel monoclonal antibodies that offer new avenues for cancer treatment. These efforts are directed towards providing medical professionals with alternative and effective therapeutic options for patients.

Escient Pharmaceuticals, Inc. is a biotechnology company based in San Diego, California, specializing in the development and manufacture of G protein-coupled receptor (GPCR)-targeted drugs aimed at treating neuro-immuno-inflammatory and autoreactive diseases. Founded in 2017, Escient focuses on harnessing the therapeutic potential of specific orphan GPCRs, particularly the Mas-Related G-Protein Receptors (Mrgprs). The company aims to provide innovative therapies for a variety of conditions, including mast cell-mediated disorders and cholestatic pruritus, thereby expanding treatment options for healthcare providers facing these complex diseases.

MBX Biosciences, Inc. is a biotechnology company founded in 2018 and based in Carmel, Indiana. The company specializes in developing therapeutics for rare endocrine disorders, focusing on conditions that currently lack effective treatments. MBX Biosciences aims to discover, develop, and commercialize first-in-class therapies, particularly peptides designed to address endocrine genetic diseases that affect glandular hormone production and the body’s chemical messaging systems.

Inversago Pharma Inc. is a preclinical-stage biotechnology company based in Montreal, Canada, specializing in the development of peripherally-restricted CB1 receptor inverse agonists aimed at treating various metabolic and fibrotic disorders. Founded in 2015, the company focuses on creating innovative therapies for conditions such as Prader-Willi syndrome, diabetes, and complications related to obesity. Its pipeline includes treatments for diabetic kidney disease, non-alcoholic steatohepatitis, and progressive fibrosis, including interstitial lung disease. Inversago Pharma aims to improve patient outcomes by offering new therapeutic options that target the underlying mechanisms of these diseases.

Gossamer Bio is a clinical-stage biopharmaceutical company based in San Diego that specializes in the discovery, development, and commercialization of innovative therapeutic products targeting significant unmet medical needs. Founded by former executives from Receptos, the company focuses on areas such as immunology, inflammation, fibrosis, and oncology. Its pipeline includes several promising candidates: Seralutinib, aimed at addressing pulmonary arterial hypertension; GB004, for inflammatory bowel disease; GB1275, targeting various oncology indications; and GB001, designed for moderate-to-severe eosinophilic asthma. Gossamer Bio's strategy involves leveraging a rich in-licensing environment to enhance its therapeutic offerings, supported by a team recognized for their successful track record in drug development.

X4 Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of innovative therapeutics for primary immune deficiencies and cancer. The company's lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4, currently undergoing multiple clinical trials. These include a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as Phase Ib and Phase IIa trials focused on severe congenital neutropenia and clear cell renal cell carcinoma, respectively. In addition, X4 Pharmaceuticals is advancing X4P-002, targeting glioblastoma multiforme, and X4P-003 aimed at primary immune deficiencies. The company has also established a license agreement with Abbisko Therapeutics to develop mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in March 2019.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, a targeted cancer therapy using gene-engineered allogeneic CAR-NK cells designed to eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A for treating hepatocellular carcinoma.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

Celcuity Inc. is a clinical-stage biotechnology company based in Minneapolis, Minnesota, focusing on cancer diagnosis and treatment. The company utilizes its CELsignia diagnostic platform, which analyzes living tumor cells to identify specific cellular processes that drive individual cancers. This innovative approach enables the discovery of new cancer sub-types that traditional molecular diagnostics may miss. Celcuity is developing diagnostic tests, including the CELsignia HSF test for HER2-negative breast cancer and the CELsignia MP test for various cancers, such as breast, lung, colon, ovarian, kidney, and bladder. By translating these discoveries into companion diagnostics, Celcuity aims to enhance therapeutic options for cancer patients and improve treatment outcomes, while also aiding pharmaceutical companies in defining targeted therapy patient populations more accurately. Founded in 2011, Celcuity is committed to advancing oncology through precise diagnostic and therapeutic innovations.

Splice Bio is a biotechnology company based in Barcelona, Spain, that specializes in developing innovative gene therapies using its proprietary intein platform technology. Founded in 2012, the company focuses on addressing the needs of patients suffering from incurable genetic diseases. Splice Bio's technology, originally developed in the Muir Lab at Princeton University, enhances the capabilities of traditional adeno-associated viruses (AAVs) by allowing for larger gene cargo delivery and targeting a broader range of tissues. This advancement aims to overcome existing limitations in gene therapy, making it a promising solution for treating complex genetic disorders. The company was formerly known as Proteodesign, S.L. before rebranding to Splice Bio in September 2020.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Sorriso Pharmaceuticals is a biotechnology company focused on developing disease-modifying antibodies aimed at treating inflammatory diseases such as Crohn's disease and ulcerative colitis. Founded in 2020 and headquartered in San Diego, California, Sorriso's innovative platform generates potent antibodies that can be administered orally, allowing for sustained activity throughout the intestinal system. This oral delivery method offers a significant advantage by alleviating the treatment burden associated with injectable antibody therapeutics, thus improving patient convenience and compliance. Through its research and development efforts, Sorriso Pharmaceuticals aims to provide effective solutions for individuals suffering from these challenging gastrointestinal conditions.

AviadoBio is a biotechnology company focused on developing transformative gene therapies for neurodegenerative disorders, specifically targeting diseases such as frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The company leverages innovative research from King’s College London and the UK Dementia Research Institute to create its therapeutic platform. By integrating a deep understanding of brain biology with proprietary gene therapies, AviadoBio aims to effectively address the challenges of delivering treatments to the appropriate areas within the nervous system. The ultimate goal is to enhance the therapeutic potential of gene therapy, potentially halting or even reversing the progression of these debilitating conditions, thereby improving the quality of life for affected individuals.

Tune Therapeutics is a biotechnology company focused on developing innovative cell and gene therapies. It employs an epigenetic programming platform to create novel therapeutic pathways, aiming to treat severe diseases without modifying the underlying DNA sequence.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company specializing in the discovery and development of microRNA-targeted therapies for various diseases. Founded in 2007 and headquartered in San Diego, California, the company focuses on innovative treatments for conditions such as kidney diseases and viral infections. Its lead product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, and RGLS4326, an anti-miR targeting miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. Additionally, Regulus is advancing a pipeline of preclinical drug candidates, including RGLS5579 to inhibit miR-10b, as well as programs aimed at treating Hepatitis B virus and Non-Alcoholic Steatohepatitis. The company's work leverages the recent scientific advancements in microRNA research, which play a crucial role in regulating gene expression.

Recludix Pharma is a biotechnology company focused on drug discovery, specifically developing potent and selective inhibitors for challenging protein targets associated with cancer and autoimmune diseases. The company utilizes a unique platform that integrates custom-generated DNA-encoded libraries, massively parallel determination of structure-activity relationships, and proprietary screening tools. This innovative approach aims to enhance the identification and development of small molecule medicines, allowing medical professionals to create precision therapies tailored to specific protein targets. By addressing complex biological challenges, Recludix Pharma strives to advance treatment options in the healthcare sector.

858 Therapeutics is a biotechnology company dedicated to the discovery of small molecule therapeutics aimed at treating cancers and other diseases, particularly those resistant to existing therapies. The company employs innovative strategies in areas such as synthetic lethality, innate immunity, and RNA modulation to develop its therapeutic candidates. Its lead program focuses on a PARG inhibitor, which is currently in early clinical development. By leveraging its platform to investigate the role of RNA modifying proteins in disease biology, 858 Therapeutics is generating a pipeline of targeted small molecules that address critical unmet medical needs in oncology and immunology.

Shape Therapeutics, Inc. is a biotechnology company focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic disorders. Founded in 2018 and headquartered in Seattle, Washington, the company utilizes its proprietary RNAfix technology to edit RNA using the body’s natural cellular machinery. This approach minimizes risks associated with immunogenicity, cellular toxicity, and unintended DNA editing. Shape Therapeutics is dedicated to advancing its innovative platforms for payload, delivery, and manufacturing to enable the development of effective treatments, aiming to address critical health challenges and improve patient outcomes.

Stablix Therapeutics is a biotechnology company specializing in Targeted Protein Stabilization (TPS) to enhance disease treatment. The company has developed a platform that produces heterobifunctional small molecules, known as RESTORACS, which selectively recruit deubiquitinase enzymes. These enzymes remove ubiquitin from specific proteins, thereby stabilizing or increasing their levels and activity. Stablix is initially applying this innovative technology to create therapeutic programs aimed at addressing rare diseases, cancer, and immunological disorders, with the potential to improve patient outcomes in these challenging areas.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

Code Biotherapeutics is a next-generation gene therapy company pioneering the development of targeted non-viral gene therapies to treat serious and life-threatening genetic diseases. Code Bio leverages its novel multivalent synthetic DNA delivery platform, 3DNA, which has been engineered to overcome many of the challenges inherent with viral-based gene therapies such as immunogenicity, size and delivery limitations, re-dosability, and manufacturing complexity. The company is advancing an internal pipeline focused on select rare disease programs as well as establishing partnerships to take forward programs in both rare and prevalent diseases.

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

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing innovative therapeutics for oncology and infectious diseases, with a particular emphasis on targeting dysfunctional lipid metabolism pathways. The company has a strong focus on fatty acid synthase (FASN) biology and has developed a pipeline of proprietary FASN inhibitors. Its lead product candidate, denifanstat, is an oral selective FASN inhibitor being investigated for the treatment of nonalcoholic steatohepatitis (NASH). Additionally, Sagimet is exploring antiviral therapeutic candidates aimed at treating respiratory syncytial virus, cytomegalovirus, and other human pathogens. Founded in 2006, Sagimet Biosciences was previously known as 3-V Biosciences, Inc. before rebranding in August 2019.

Personal Genome Diagnostics, Inc. is a biotechnology company focused on patient-specific analyses of cancer genomes. Established in 2010 and headquartered in Baltimore, Maryland, the company has pioneered technologies for non-invasive "liquid biopsy" approaches, allowing for the detection of tumor-specific alterations in circulating cell-free DNA. Its offerings include the METDetect Assay for identifying MET gene amplifications, LungSelect for detecting actionable genetic alterations in non-small cell lung cancer patients, and PGDx elio plasma resolve, which analyzes various genetic mutations from plasma samples. Additionally, Personal Genome Diagnostics provides RNAcomplete, enabling researchers to extract both RNA and genomic DNA from a single tissue sample, and CancerXOME, which captures and analyzes coding regions of genes. The company collaborates with institutions like Mayo Clinic and partners with KingMed Diagnostics to enhance its research and diagnostic capabilities, thereby empowering oncologists and researchers with crucial genomic insights to combat cancer.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, a targeted cancer therapy using gene-engineered allogeneic CAR-NK cells designed to eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A for treating hepatocellular carcinoma.

Resilience, established in 2020 and headquartered in La Jolla, California, is a biopharmaceutical manufacturing and technology company. It specializes in providing end-to-end manufacturing and development solutions for complex medicines, including biologics, vaccines, nucleic acids, and cell and gene therapies. Resilience invests in developing new manufacturing technologies to ensure rapid, safe, and scalable production, thereby enhancing patient access to these treatments. The company partners with biopharmaceutical companies to focus on breakthrough discoveries while Resilience manages the manufacturing process, safeguarding supply chains against future disruptions.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Polyneuron Pharmaceuticals AG is a biopharmaceutical company based in Basel, Switzerland, focused on developing innovative therapeutics for the treatment of autoimmune disorders. Founded in 2014, the company specializes in creating drugs to target specific autoimmune conditions, including PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron's therapeutic approach employs biodegradable glycopolymers that selectively eliminate pathological autoantibodies, preserving the overall integrity of the immune system. This technology, referred to as Antibody-Catch, aims to provide safe and effective treatment options for patients suffering from autoimmune diseases affecting the peripheral nervous system, particularly those involving autoantibodies against carbohydrate epitopes.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

MBX Biosciences, Inc. is a biotechnology company founded in 2018 and based in Carmel, Indiana. The company specializes in developing therapeutics for rare endocrine disorders, focusing on conditions that currently lack effective treatments. MBX Biosciences aims to discover, develop, and commercialize first-in-class therapies, particularly peptides designed to address endocrine genetic diseases that affect glandular hormone production and the body’s chemical messaging systems.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Amplyx Pharmaceuticals, Inc. is a small molecule drug development company focused on creating innovative therapies for debilitating and life-threatening diseases, particularly those affecting individuals with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in the development of oral and injectable anti-infective drugs, including antifungal agents designed to treat invasive fungal infections caused by pathogens such as Candida and Aspergillus. Amplyx employs a unique platform approach that enhances the efficacy and reduces the toxicity of existing drugs by incorporating a second small molecule. This strategy aims to provide effective treatment options for patients undergoing chemotherapy and other immunosuppressive therapies, ensuring they receive appropriate care both during hospitalization and after discharge.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for the treatment of severe diseases affecting the muscle and central nervous system (CNS). Established in 2019 and based in Waltham, Massachusetts, the company specializes in creating adeno-associated virus (AAV) vectors and utilizes a platform that integrates synthetic and systems biology with high-throughput screening techniques. This innovative approach allows Affinia Therapeutics to design novel gene therapies with enhanced properties tailored to address significant unmet medical needs. The company aims to provide healthcare professionals with advanced therapeutic options for patients suffering from debilitating conditions.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

Geron Corporation is a late-stage clinical biopharmaceutical company based in Menlo Park, California, specializing in the development and commercialization of therapeutics for hematologic malignancies. Established in 1990, the company is primarily focused on its investigational drug, imetelstat, a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. Imetelstat targets the uncontrolled proliferation of malignant progenitor cells, aiming to improve blood cell production in patients with conditions such as myelodysplastic syndromes and myelofibrosis. Geron generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements, positioning itself as a key player in oncology therapeutics.

ERYTECH Pharma S.A. is a clinical-stage biopharmaceutical company based in Lyon, France, founded in 2004. The company specializes in the development of red blood cell-based therapeutics aimed at treating cancer and orphan diseases. Its lead product candidate, eryaspase, is currently undergoing Phase III clinical trials for second-line pancreatic cancer and Phase II trials for triple-negative breast cancer. Additionally, ERYTECH is developing erymethionase, a preclinical candidate targeting methionine-dependent cancers, which consists of methionine-gamma-lyase encapsulated in red blood cells. The company collaborates with research institutions such as the Fox Chase Cancer Center and Queen’s University of Canada to enhance the preclinical development of its products for conditions like homocystinuria and arginase-1 deficiency, respectively. Through its innovative approach to encapsulating therapeutic molecules, ERYTECH Pharma aims to improve the efficacy and safety of its treatments.

ChromaCode Inc., established in 2012 and based in Carlsbad, California, specializes in molecular diagnostics. The company develops innovative testing solutions for infectious diseases, cancer, and non-invasive prenatal testing (NIPT) using technology licensed from Caltech. ChromaCode's core products are software, reagents, and assays that enhance the multiplexing capabilities of quantitative PCR (qPCR) instruments by fivefold. Additionally, they offer molecular assays and software for various applications such as personalized medicine and pharmaceutical research. One of their notable products is the HDPCR Tick-Borne Pathogen Panel, a multiplex PCR test detecting common tick-borne pathogens in a single assay. ChromaCode's technology focuses on improving signal processing from life science instrumentation, enabling cost-effective testing of multiple targets simultaneously while integrating into existing laboratory workflows.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

Shape Therapeutics, Inc. is a biotechnology company focused on RNA-editing gene therapy for various medical conditions, including neurodegenerative disorders, oncology, metabolic diseases, and rare genetic disorders. Founded in 2018 and headquartered in Seattle, Washington, the company utilizes its proprietary RNAfix technology to edit RNA using the body’s natural cellular machinery. This approach minimizes risks associated with immunogenicity, cellular toxicity, and unintended DNA editing. Shape Therapeutics is dedicated to advancing its innovative platforms for payload, delivery, and manufacturing to enable the development of effective treatments, aiming to address critical health challenges and improve patient outcomes.

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

Inventiva Pharma is a clinical-stage biopharmaceutical company based in France, dedicated to developing oral small molecule therapies aimed at addressing significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company has established a robust pipeline supported by a proprietary discovery engine and a wholly-owned research and development facility. It possesses an extensive library of molecules and a team with considerable expertise in developing compounds that target nuclear receptors, transcription factors, and epigenetic modulation. Inventiva is advancing clinical candidates, including lanifibranor, which is being developed for non-alcoholic steatohepatitis (NASH), a condition currently lacking approved treatments. Additionally, the company is engaged in a variety of pre-clinical therapy programs, further strengthening its portfolio in the fields of oncology, fibrosis, and rare diseases.

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

Sagimet Biosciences is a clinical-stage biopharmaceutical company based in San Mateo, California, that specializes in developing innovative therapeutics for oncology and infectious diseases, with a particular emphasis on targeting dysfunctional lipid metabolism pathways. The company has a strong focus on fatty acid synthase (FASN) biology and has developed a pipeline of proprietary FASN inhibitors. Its lead product candidate, denifanstat, is an oral selective FASN inhibitor being investigated for the treatment of nonalcoholic steatohepatitis (NASH). Additionally, Sagimet is exploring antiviral therapeutic candidates aimed at treating respiratory syncytial virus, cytomegalovirus, and other human pathogens. Founded in 2006, Sagimet Biosciences was previously known as 3-V Biosciences, Inc. before rebranding in August 2019.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Polyneuron Pharmaceuticals AG is a biopharmaceutical company based in Basel, Switzerland, focused on developing innovative therapeutics for the treatment of autoimmune disorders. Founded in 2014, the company specializes in creating drugs to target specific autoimmune conditions, including PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron's therapeutic approach employs biodegradable glycopolymers that selectively eliminate pathological autoantibodies, preserving the overall integrity of the immune system. This technology, referred to as Antibody-Catch, aims to provide safe and effective treatment options for patients suffering from autoimmune diseases affecting the peripheral nervous system, particularly those involving autoantibodies against carbohydrate epitopes.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

Oyster Point Pharma, Inc., established in 2015 and headquartered in Princeton, New Jersey, is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for ocular surface diseases. Its lead product candidate, OC-01, is a nasal spray formulated as a highly selective nicotinic acetylcholine receptor (nAChR) agonist, currently in Phase III trials for treating the signs and symptoms of dry eye disease, a chronic condition affecting over 30 million Americans. OC-01's novel mechanism of action stimulates tear production by activating the trigeminal parasympathetic pathway, re-establishing tear film homeostasis.

Black Diamond Therapeutics, Inc., established in 2014 and headquartered in Cambridge, Massachusetts, specializes in precision oncology medicine. The company discovers and develops small molecule therapies that are tumor-agnostic, meaning they target specific genetic mutations across various cancer types rather than being limited to a single cancer indication. Its lead product candidate, BDTX-189, inhibits non-canonical and oncogenic mutations of ErbB kinases epidermal growth factor receptor (EGFR). The company's Mutation-Allostery-Pharmacology platform enables it to target undrugged mutations in patients with genetically defined cancers, offering new treatment options where few exist. Black Diamond Therapeutics is also advancing early-stage pipeline programs targeting allosteric mutations in other cancer-related kinases.

Tiburio Therapeutics, founded in 2018 and based in Cambridge, Massachusetts, specializes in developing and manufacturing medicines for rare endocrine diseases and tumors related to the endocrine system. The company's primary focus is on TBR-760, a drug candidate designed to treat non-functioning pituitary adenoma (NFPA) using a dopamine-somatostatin chimeric molecule aimed at shrinking or halting tumor growth. Additionally, Tiburio explores other compounds like TBR-065 for treating rare endocrine diseases where effective treatments are limited.

Curzion Pharmaceuticals is a privately held development-stage biopharma company, and its development-stage oral selective lysophosphatidic acid 1 receptor (LPAR1) antagonist, CZN001 (renamed HZN-825).

Xenon Pharmaceuticals Inc. is a clinical-stage biopharmaceutical company based in Burnaby, Canada, focused on developing innovative therapeutics for neurological disorders, including rare central nervous system conditions. The company's pipeline includes XEN496 and XEN1101, both Kv7 potassium channel modulators currently in Phase II clinical trials for the treatment of epilepsy. Additionally, Xenon is advancing XEN901, a selective Nav1.6 sodium channel inhibitor, and XEN007, a central nervous system-acting calcium channel modulator, both in Phase I clinical trials. Through its proprietary discovery platform known as Extreme Genetics, Xenon leverages genetic insights from families with severe phenotypes to identify and characterize single-gene defects that may serve as drug targets. The company has also established a collaboration agreement with Neurocrine Biosciences to develop first-in-class treatments for epilepsy, highlighting its commitment to addressing both rare and more prevalent neurological diseases.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Mirum Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing innovative therapies for debilitating liver diseases. Founded in 2018 and headquartered in Foster City, California, Mirum's lead product candidate is maralixibat, an investigational oral drug currently in Phase 3 clinical trials targeting progressive familial intrahepatic cholestasis, Alagille syndrome, and biliary atresia. The company’s approved product, Livmarli, is aimed at alleviating cholestatic pruritus in patients with Alagille syndrome. In addition to maralixibat, Mirum is also advancing the development of volixibat, which is intended for treating intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Through its focused pipeline, Mirum aims to address significant unmet medical needs in rare and orphan liver diseases.

Qpex Biopharma is a San Diego-based biopharmaceutical company founded in 2018, focused on developing innovative therapies to address the growing challenge of antimicrobial resistance. The company specializes in creating a pipeline of antibiotic drugs aimed at treating infectious diseases in both inpatient and outpatient settings. With a strong emphasis on the discovery, development, and regulatory approval of anti-infective medicines, Qpex Biopharma leverages its expertise and experience in collaboration with public and private partnerships to meet critical medical needs. The company's mission is to provide effective solutions for healthcare professionals facing the complexities of resistant infections, thereby enhancing treatment options for patients.

PhaseBio Pharmaceuticals, Inc., established in 2002 and based in Malvern, Pennsylvania, is a clinical-stage biopharmaceutical company dedicated to developing novel therapies for cardiopulmonary diseases. Its primary focus is on PB2452, a reversal agent for the antiplatelet drug ticagrelor, currently in Phase III trials for managing uncontrolled bleeding events or urgent surgeries. Additionally, PhaseBio is developing PB1046, a fusion protein in Phase IIb trials for treating pulmonary arterial hypertension, and PB6440, an oral agent for resistant hypertension. The company's proprietary technology, elastin-like polypeptides (ELPs), enhances the stability, bioavailability, and efficacy of proteins and peptides, aiming to improve patient outcomes and reduce side effects.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Millendo Therapeutics is a biopharmaceutical company dedicated to developing innovative treatments for endocrine diseases resulting from hormone dysregulation. The company focuses on creating distinct and transformative therapies that address significant unmet medical needs in this area. By leveraging scientific advancements, Millendo aims to develop novel compounds that can effectively treat complex conditions, ultimately enhancing clinical care and improving the quality of life for patients, families, and caregivers.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

Catalog Technologies Inc. specializes in a DNA-based platform that revolutionizes digital data storage and computation. Founded in 2016 and headquartered in Boston, Massachusetts, the company provides a method for encoding digital information into DNA, making it a viable and cost-effective solution for long-term data archiving. By leveraging advanced synthetic biology technologies, Catalog aims to transform how information is stored, allowing customers to utilize DNA molecules as a primary medium for digital records. This innovative approach positions Catalog at the forefront of next-generation data storage solutions.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, established in 2014. It specializes in the discovery and development of innovative therapies aimed at treating liver and gastrointestinal diseases. The company is advancing a pipeline of drug candidates, with a primary focus on the farnesoid X receptor (FXR), a key target in managing various liver and GI disorders. Its lead candidates, MET409 and MET642, are currently in clinical trials to evaluate their efficacy in treating non-alcoholic steatohepatitis. Additionally, Metacrine collaborates with Novo Nordisk A/S to explore further research opportunities related to fibroblast growth factor 1. The company is committed to developing best-in-class therapies to improve patient outcomes in these challenging medical areas.

Tempus, established in 2015 and based in Chicago, Illinois, is a technology company specializing in precision medicine. It develops a comprehensive library of molecular and clinical data, along with an operating system that makes this information accessible and useful for patients, physicians, and researchers. Tempus offers services such as genomic sequencing, clinical data restructuring, and biological modeling, enabling real-time data-driven decisions for personalized patient care and facilitating the discovery of optimized therapeutic options.

Senti Bio, established in 2016 and headquartered in South San Francisco, specializes in synthetic biotechnology for therapeutic development. The company's core focus is engineering gene circuits, a platform technology that enables precise control over cellular behavior. Senti Bio's innovative approach allows for the creation of therapies with enhanced specificity and control, particularly in oncology. Their product pipeline includes SENTI-202, a targeted cancer therapy using gene-engineered allogeneic CAR-NK cells designed to eliminate cancer cells while sparing healthy bone marrow, and SENTI-301A for treating hepatocellular carcinoma.

Liquidia Technologies is a late-stage clinical biopharmaceutical company based in the United States, dedicated to developing and commercializing innovative therapeutics. Utilizing its proprietary PRINT technology, a particle engineering platform, Liquidia produces uniform drug particles aimed at enhancing the safety and efficacy of various therapies. The company is advancing two key product candidates: LIQ861, intended for the treatment of pulmonary arterial hypertension, and LIQ865, designed for managing local post-operative pain. Additionally, Liquidia collaborates with leading pharmaceutical firms to leverage its PRINT technology across multiple therapeutic areas, molecule types, and administration routes, addressing unmet patient needs, particularly in the realm of pulmonary hypertension.

Xoc Pharmaceuticals, Inc. is a biotechnology company based in Los Gatos, California, that focuses on developing innovative therapeutics for neurology and related therapeutic areas. Incorporated in 2013, the company employs advanced receptor characterization technology and cutting-edge medicinal chemistry to identify and develop novel oral therapies aimed at preventing migraines and managing Parkinson's disease. Xoc Pharmaceuticals emphasizes improved safety and efficacy in its treatments, utilizing a development strategy that incorporates targeted molecular modifications and optimized delivery routes. By leveraging the pharmacological history of compounds similar to its product candidates, the company effectively manages clinical and regulatory risks associated with its development programs, ultimately striving to enhance patient outcomes for those with neurological disorders.

CTI BioPharma Corp. is a biopharmaceutical company headquartered in Seattle, Washington, that specializes in the acquisition, development, and commercialization of targeted therapies for blood-related cancers. Founded in 1991 and formerly known as Cell Therapeutics, Inc., the company is primarily focused on developing novel treatments to improve patient outcomes in oncology. Its key investigational product, pacritinib, is an oral kinase inhibitor currently in Phase III clinical trials for the treatment of myelofibrosis in adults. In addition to pacritinib, CTI BioPharma's portfolio includes other candidates like Pixantrone, which is undergoing Phase III trials for non-Hodgkin's lymphoma, and Brostallicin, currently in a Phase II study for sarcoma. The company has established collaboration and licensing agreements with several pharmaceutical partners.

Personal Genome Diagnostics, Inc. is a biotechnology company focused on patient-specific analyses of cancer genomes. Established in 2010 and headquartered in Baltimore, Maryland, the company has pioneered technologies for non-invasive "liquid biopsy" approaches, allowing for the detection of tumor-specific alterations in circulating cell-free DNA. Its offerings include the METDetect Assay for identifying MET gene amplifications, LungSelect for detecting actionable genetic alterations in non-small cell lung cancer patients, and PGDx elio plasma resolve, which analyzes various genetic mutations from plasma samples. Additionally, Personal Genome Diagnostics provides RNAcomplete, enabling researchers to extract both RNA and genomic DNA from a single tissue sample, and CancerXOME, which captures and analyzes coding regions of genes. The company collaborates with institutions like Mayo Clinic and partners with KingMed Diagnostics to enhance its research and diagnostic capabilities, thereby empowering oncologists and researchers with crucial genomic insights to combat cancer.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Pionyr Immunotherapeutics Inc. is a biotechnology company focused on developing innovative cancer immunotherapies that specifically target the tumor microenvironment to enhance the body's antitumor immunity. Founded in 2015 and based in San Francisco, California, Pionyr leverages advanced target discovery and antibody generation platform technologies to create next-generation immuno-oncology therapeutics. The company's proprietary approach, known as Myeloid Tuning™, aims to improve the immune system’s anti-tumor response by precisely altering the cellular composition within the tumor microenvironment. This strategy is designed to enable patients to better combat cancer, particularly when used in conjunction with checkpoint inhibitors.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, established in 2014. It specializes in the discovery and development of innovative therapies aimed at treating liver and gastrointestinal diseases. The company is advancing a pipeline of drug candidates, with a primary focus on the farnesoid X receptor (FXR), a key target in managing various liver and GI disorders. Its lead candidates, MET409 and MET642, are currently in clinical trials to evaluate their efficacy in treating non-alcoholic steatohepatitis. Additionally, Metacrine collaborates with Novo Nordisk A/S to explore further research opportunities related to fibroblast growth factor 1. The company is committed to developing best-in-class therapies to improve patient outcomes in these challenging medical areas.

Akouos, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

Inozyme Pharma, Inc. is a biopharmaceutical company focused on developing therapies for rare diseases related to abnormal mineralization affecting the vasculature, soft tissue, and skeletal systems. The company's lead product candidate, INZ-701, is a recombinant protein designed to treat rare genetic conditions caused by ENPP1 and ABCC6 deficiencies, as well as calciphylaxis. Inozyme's therapies aim to address the underlying causes of these debilitating diseases, providing potentially disease-modifying treatments for conditions such as Generalized Arterial Calcification of Infancy and Autosomal Recessive Hypophosphatemic Rickets Type 2. The company has a licensing agreement with Yale University for certain therapeutic applications and is headquartered in Boston, Massachusetts. Established in 2015, Inozyme is dedicated to improving the quality of life for patients affected by severe metabolic disorders associated with mineral imbalances.

Oyster Point Pharma, Inc., established in 2015 and headquartered in Princeton, New Jersey, is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for ocular surface diseases. Its lead product candidate, OC-01, is a nasal spray formulated as a highly selective nicotinic acetylcholine receptor (nAChR) agonist, currently in Phase III trials for treating the signs and symptoms of dry eye disease, a chronic condition affecting over 30 million Americans. OC-01's novel mechanism of action stimulates tear production by activating the trigeminal parasympathetic pathway, re-establishing tear film homeostasis.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The company identifies and de-risks compounds that hold therapeutic and commercial potential, employing a rigorous process to generate data that supports both development pathways and the formation of new companies. With a team that boasts deep expertise in drug development and strong relationships within academia and patient advocacy, Cydan accelerates therapies through various stages, including clinical, regulatory, and commercial development. By facilitating the creation of effective treatments, Cydan aims to improve the lives of patients and families affected by rare diseases.