New Enterprise Associates

Abcuro develops immunotherapies targeting autoimmunity and cancer. Founded in 2015, the company uses bioinformatics and ex-vivo validation to uncover novel immune system targets.

Tune Therapeutics develops cell and gene therapies guided by epigenetic programming to treat devastating diseases. The company is building an epigenetic programming platform that enables new pathways for disease treatment by tapping into the epigenome to program biology without altering the DNA sequence. This approach aims to deliver paradigm-changing therapies by modulating gene expression through epigenetic mechanisms rather than editing the genome, expanding options for diseases where conventional treatments have limited efficacy. By integrating epigenetics and cell biology, the company seeks durable, precise interventions that can reprogram cellular states to restore function while minimizing genetic change. The emphasis is on research and development to advance candidates toward clinical use to address severe and unmet medical needs.

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.

FIRE1 is an Irish-based company specializing in the development of innovative therapeutic devices. Its flagship product is a remote monitoring system designed to enhance heart failure patients' lives by providing real-time data to healthcare providers, facilitating early detection and proactive interventions.

Orbis Medicines is a company that develops a macrocyclic chemistry and computational platform for oral macrocycle drug discovery. The platform generates macrocycle candidates, nCycles, optimized for oral bioavailability to advance therapeutics that historically faced development hurdles. Its pipeline centers on nCycle candidates targeting validated biologic targets that are typically addressed by injectable drugs, with the goal of enabling oral delivery of such therapies. The platform combines chemistry and computational methods to accelerate drug discovery toward challenging targets in unmet-need indications, supporting therapeutics designed to improve patient outcomes.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Avenzo Therapeutics focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

Pathos develops an AI-enabled drug discovery platform aimed at reducing the medication development process. Its self-learning platform uses big data, technology, and artificial intelligence to identify targets, validate them, and establish their clinical efficacy, enabling drug developers to explore novel concepts and advance breakthrough therapies for various diseases.

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 specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

Moximed develops and provides the Atlas System, an implantable joint unloader designed to alleviate pain and preserve mobility in patients with medial compartment knee osteoarthritis. Its outpatient treatment option aims to improve quality of life for those not responsive to other therapies or candidates for knee replacement.

MBX Biosciences is a biotechnology company focused on developing therapies for endocrine and metabolic disorders, emphasizing rare endocrine diseases with limited treatment options. It pursues discovery, development, and commercialization of first-in-class peptide therapeutics that modulate glandular hormone signaling. MBX has developed a proprietary Precision Endocrine Peptide platform to engineer peptides with extended time-action profiles and consistent drug exposure, aiming to overcome limitations of traditional peptide therapies and improve disease management. Based in Carmel, Indiana, the company advances novel peptide programs through clinical development to address unmet medical needs in endocrine and metabolic disorders.

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 specializes in developing advanced bioelectronic therapies that can sense and adapt, aiming to enhance clinical outcomes for patients with functional disorders of the peripheral nervous system.

Zenas BioPharma is a clinical-stage biopharmaceutical company focused on developing immune-based therapies for autoimmune and immune-mediated diseases. Based in Florida, United States, the company advances biologic programs and aims to modulate B cell–driven pathology. Its lead product candidate, obexelimab, is a bifunctional monoclonal antibody designed to bind both CD19 and FcγRIIb on B cells, thereby inhibiting harmful immune activity without depleting B cells.

Xaira Therapeutics is a biotechnology company that leverages artificial intelligence to revolutionize drug discovery and development. Headquartered in the San Francisco Bay Area, Xaira brings together machine learning experts, expansive data generation, and robust therapeutic product development to advance treatments for various diseases.

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 focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

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 is a biopharmaceutical company developing an investigational siRNA medicine for preterm preeclampsia. Its mission is to provide safe, effective, evidence-based, and affordable therapies for treating life-threatening complications of pregnancy.

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.

FIRE1 is an Irish-based company specializing in the development of innovative therapeutic devices. Its flagship product is a remote monitoring system designed to enhance heart failure patients' lives by providing real-time data to healthcare providers, facilitating early detection and proactive interventions.

Launchpad Therapeutics is an antibody-focused precision oncology company.

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 is a biotechnology company focused on developing therapies for endocrine and metabolic disorders, emphasizing rare endocrine diseases with limited treatment options. It pursues discovery, development, and commercialization of first-in-class peptide therapeutics that modulate glandular hormone signaling. MBX has developed a proprietary Precision Endocrine Peptide platform to engineer peptides with extended time-action profiles and consistent drug exposure, aiming to overcome limitations of traditional peptide therapies and improve disease management. Based in Carmel, Indiana, the company advances novel peptide programs through clinical development to address unmet medical needs in endocrine and metabolic disorders.

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.

Allakos is a clinical-stage biopharmaceutical company developing antibody-based therapeutics targeting immunomodulatory receptors on immune effector cells. Its pipeline focuses on allergic, inflammatory, and proliferative diseases, with lead programs targeting Siglec-6 (AK006) and Siglec-8.

Moximed develops and provides the Atlas System, an implantable joint unloader designed to alleviate pain and preserve mobility in patients with medial compartment knee osteoarthritis. Its outpatient treatment option aims to improve quality of life for those not responsive to other therapies or candidates for knee replacement.

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 is a clinical-stage biopharmaceutical company focused on developing novel oral small molecule therapeutics targeting chemokine receptor CXCR4. Its lead drug candidate, mavorixafor (X4P-001), is in Phase III trials for the treatment of WHIM syndrome and has shown promising results in other indications such as severe congenital neutropenia and clear cell renal cell carcinoma.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

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 is a clinical-stage biotechnology company that discovers new cancer sub-types and therapeutic options using its proprietary CELsignia diagnostic platform. This platform analyzes living tumor cells to identify specific abnormal cellular processes driving cancer, enabling the development of targeted therapies for patients.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, established in 2011. The company specializes in the development and commercialization of Haduvio, an oral extended-release formulation of nalbuphine. Haduvio is currently undergoing a Phase IIb/III clinical trial, known as the PRISM trial, aimed at treating chronic pruritus associated with prurigo nodularis. Additionally, Trevi is exploring the use of nalbuphine ER for other serious neurologically mediated conditions, including chronic cough in patients with idiopathic pulmonary fibrosis and levodopa-induced dyskinesia in individuals with Parkinson’s disease. The treatment targets conditions that share a common pathophysiology mediated through opioid receptors in the central and peripheral nervous systems.

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.

Founded in 2020 and headquartered in San Diego, California, Sorriso Pharmaceuticals is a biotechnology company dedicated to developing disease-modifying antibodies for treating inflammatory diseases such as Crohn's disease and ulcerative colitis. Its proprietary platform generates potent antibodies designed for oral delivery, aiming to maintain therapeutic activity throughout the intestinal system.

Tune Therapeutics develops cell and gene therapies guided by epigenetic programming to treat devastating diseases. The company is building an epigenetic programming platform that enables new pathways for disease treatment by tapping into the epigenome to program biology without altering the DNA sequence. This approach aims to deliver paradigm-changing therapies by modulating gene expression through epigenetic mechanisms rather than editing the genome, expanding options for diseases where conventional treatments have limited efficacy. By integrating epigenetics and cell biology, the company seeks durable, precise interventions that can reprogram cellular states to restore function while minimizing genetic change. The emphasis is on research and development to advance candidates toward clinical use to address severe and unmet medical needs.

AviadoBio focuses on developing transformative gene therapies for devastating neurodegenerative disorders such as frontotemporal dementia and amyotrophic lateral sclerosis. The company's platform combines proprietary technologies with a deep understanding of the brain to deliver effective treatments.

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 develops a platform for discovering potent and selective inhibitors of complex protein targets. The platform integrates custom-generated DNA-encoded libraries, massively parallel structure-activity relationship determination, and proprietary screening tools to ensure selectivity.

858 Therapeutics is a biotechnology company specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

Allay Therapeutics is pioneering ultra-sustained pain therapeutics to transform post-surgical pain management. Its proprietary platform combines non-opioid analgesics and biopolymers to create dissolvable therapeutics delivering pain relief within targeted sites over weeks, significantly longer than current treatments. The company's lead product candidate, ATX101, is in Phase 2 clinical trials for total knee replacement surgeries.

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). It develops heterobifunctional small molecules that recruit deubiquitinase enzymes to stabilize or enhance targeted proteins, focusing on treating rare diseases, cancer, and immunological disorders.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

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.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraine. The company's lead product candidate, STS101, is a drug-device combination that incorporates a proprietary dry-powder formulation of dihydroergotamine mesylate. Designed for self-administration, STS101 utilizes a pre-filled, single-use nasal delivery device, enhancing patient convenience and accessibility. The product is currently undergoing Phase III clinical trials, marking a significant step in its development. Founded in 2016, Satsuma is headquartered in South San Francisco, California.

Sagimet Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapeutics to treat diseases like nonalcoholic steatohepatitis (NASH) and certain cancers. The company specializes in targeting dysfunctional metabolic pathways, with a particular expertise in fatty acid synthase (FASN) biology.

Spyglass Ophthalmics, Inc. is a company based in Mission Viejo, California, specializing in the development of ophthalmic therapeutic devices. Founded in 2019, Spyglass focuses on creating innovative solutions to enhance vision and eye care for patients. The company's products target unmet needs in areas such as cataract treatment, lens replacement, and other chronic ophthalmic diseases. By providing these devices, Spyglass aims to ensure that patients receive quality eye treatment at an affordable cost.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in San Diego, California, dedicated to developing therapies for patients with rare genetic mitochondrial diseases. These conditions are characterized by deficits in cellular metabolism and energy production, often leading to significant health challenges. The company is focused on creating treatments that improve mitochondrial function, preserve muscle integrity, and enhance the overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes critical to the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. Founded in 2014, Reneo aims to address the unmet needs of patients suffering from orphan metabolic diseases linked to mitochondrial dysfunction.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

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 is a biotechnology company focused on developing therapies for endocrine and metabolic disorders, emphasizing rare endocrine diseases with limited treatment options. It pursues discovery, development, and commercialization of first-in-class peptide therapeutics that modulate glandular hormone signaling. MBX has developed a proprietary Precision Endocrine Peptide platform to engineer peptides with extended time-action profiles and consistent drug exposure, aiming to overcome limitations of traditional peptide therapies and improve disease management. Based in Carmel, Indiana, the company advances novel peptide programs through clinical development to address unmet medical needs in endocrine and metabolic disorders.

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 focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

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 develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Cardionomic is a medical device company developing a catheter-based neuromodulation therapy for acute decompensated heart failure. The device delivers targeted electrical stimulation to cardiac nerves via placement in the right pulmonary artery through the internal jugular vein and modulates autonomic signals to improve ventricular contractility without increasing heart rate. The therapy aims to increase cardiac output, renal blood flow, urine production, and pharmacological tolerance while reducing deleterious hormonal signaling associated with heart failure. It addresses the root cause of ADHF by non-systemically enhancing contractility to improve in-hospital outcomes. The company was founded in 2007 and is based in New Brighton, Minnesota.

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.

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.

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.

Black Diamond Therapeutics discovers and develops small molecule therapies for precision oncology. It focuses on allosteric mutations in kinases related to cancer, with a lead product candidate targeting non-canonical ErbB kinase mutations.

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.

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 clinical-stage biopharmaceutical company focused on developing allogeneic, off-the-shelf engineered natural killer (NK) cell therapies. The company aims to harness the inherent power of NK cells to target and destroy tumor cells, offering a potent and better-tolerated alternative to traditional T-cell therapies for a broad range of cancer indications.

Sagimet Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapeutics to treat diseases like nonalcoholic steatohepatitis (NASH) and certain cancers. The company specializes in targeting dysfunctional metabolic pathways, with a particular expertise in fatty acid synthase (FASN) biology.

Vesper Medical, Inc. is a medical device company based in Wayne, Pennsylvania, founded in 2016. The company specializes in the development of minimally invasive peripheral vascular products aimed at treating patients with deep venous disease. Its primary offering is the Vesper DUO Venous Stent System, designed specifically for venous stenting. This system features a unique combination of strength and flexibility to address the complex anatomical challenges encountered in the iliac and femoral veins. Vesper Medical is dedicated to creating safe, effective, and user-friendly products that enhance patient care and support healthcare providers in improving patient outcomes in venous treatment.

Reneo Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company based in San Diego, California, dedicated to developing therapies for patients with rare genetic mitochondrial diseases. These conditions are characterized by deficits in cellular metabolism and energy production, often leading to significant health challenges. The company is focused on creating treatments that improve mitochondrial function, preserve muscle integrity, and enhance the overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes critical to the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. Founded in 2014, Reneo aims to address the unmet needs of patients suffering from orphan metabolic diseases linked to mitochondrial dysfunction.

Spyglass Ophthalmics, Inc. is a company based in Mission Viejo, California, specializing in the development of ophthalmic therapeutic devices. Founded in 2019, Spyglass focuses on creating innovative solutions to enhance vision and eye care for patients. The company's products target unmet needs in areas such as cataract treatment, lens replacement, and other chronic ophthalmic diseases. By providing these devices, Spyglass aims to ensure that patients receive quality eye treatment at an affordable cost.

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 discovers and develops small molecule therapies for precision oncology. It focuses on allosteric mutations in kinases related to cancer, with a lead product candidate targeting non-canonical ErbB kinase mutations.

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).

Founded in 2009, Lungpacer Medical is a medical device company based in Vancouver, Canada. It specializes in developing therapeutic solutions to preserve and restore the strength of the diaphragm muscle in critically ill patients requiring mechanical ventilation. The company's flagship product, the Lungpacer DPT System, uses a minimally invasive, transvascular nerve stimulation approach to activate the diaphragm, potentially improving patient outcomes and reducing hospital care costs.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, established in 2011. The company specializes in the development and commercialization of Haduvio, an oral extended-release formulation of nalbuphine. Haduvio is currently undergoing a Phase IIb/III clinical trial, known as the PRISM trial, aimed at treating chronic pruritus associated with prurigo nodularis. Additionally, Trevi is exploring the use of nalbuphine ER for other serious neurologically mediated conditions, including chronic cough in patients with idiopathic pulmonary fibrosis and levodopa-induced dyskinesia in individuals with Parkinson’s disease. The treatment targets conditions that share a common pathophysiology mediated through opioid receptors in the central and peripheral nervous systems.

Xenon Pharmaceuticals is a biopharmaceutical company dedicated to developing novel medicines for neurological disorders. It focuses on rare genetic defects as drug targets, leveraging its proprietary discovery platform called Extreme Genetics. The company's pipeline includes XEN1101 and XEN496 for epilepsy treatment.

Allay Therapeutics is pioneering ultra-sustained pain therapeutics to transform post-surgical pain management. Its proprietary platform combines non-opioid analgesics and biopolymers to create dissolvable therapeutics delivering pain relief within targeted sites over weeks, significantly longer than current treatments. The company's lead product candidate, ATX101, is in Phase 2 clinical trials for total knee replacement surgeries.

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 therapies for debilitating liver diseases. Its primary focus is on Maralixibat, an investigational oral drug in Phase 3 trials for treating progressive familial intrahepatic cholestasis disease and Alagille syndrome.

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.

Galera Therapeutics is a clinical-stage biopharmaceutical company focused on developing therapeutics to mitigate radiotherapy-induced side effects in cancer patients. Its lead product candidate, avasopasem manganese (GC4419), is a small molecule superoxide dismutase mimetic currently in Phase III trials for treating radiation-induced severe oral mucositis in head and neck cancer patients.

PhaseBio Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing novel therapies for cardiopulmonary diseases. Its lead product candidate, PB2452, is a reversal agent for the antiplatelet drug ticagrelor, intended to manage uncontrolled bleeding events or urgent surgeries. PhaseBio is also developing PB1046 for pulmonary arterial hypertension and PB6440 for resistant hypertension.

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 is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

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.

MEI Pharma is a biopharmaceutical company focused on developing novel therapies for cancer. Its pipeline includes Zandelisib (PI3K inhibitor) and Voruciclib (CDK9 inhibitor), both in clinical trials, as well as Pracinostat (HDAC inhibitor). The company collaborates with other pharmaceutical companies for drug development.

Vesper Medical, Inc. is a medical device company based in Wayne, Pennsylvania, founded in 2016. The company specializes in the development of minimally invasive peripheral vascular products aimed at treating patients with deep venous disease. Its primary offering is the Vesper DUO Venous Stent System, designed specifically for venous stenting. This system features a unique combination of strength and flexibility to address the complex anatomical challenges encountered in the iliac and femoral veins. Vesper Medical is dedicated to creating safe, effective, and user-friendly products that enhance patient care and support healthcare providers in improving patient outcomes in venous treatment.

Senti Biosciences is a biotechnology company focused on synthetic biology. It develops gene circuit platforms to engineer therapeutics, with a focus on improving precision and control in cancer treatments.

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.