New Enterprise Associates

X4 Pharmaceuticals is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing novel therapeutics for primary immune deficiencies and cancer. Its lead drug candidate, mavorixafor, is an oral small molecule antagonist of the chemokine receptor CXCR4 and is currently undergoing various clinical trials, including a Phase III trial for patients with warts, hypogammaglobulinemia, infections, and myelokathexis syndrome, as well as trials for severe congenital neutropenia and clear cell renal cell carcinoma. The company is also advancing X4P-002, which targets glioblastoma multiforme, and X4P-003, aimed at treating primary immune deficiencies. Additionally, X4 Pharmaceuticals has a collaboration with Abbisko Therapeutics to explore mavorixafor in combination with checkpoint inhibitors for oncology applications. Founded in 2014, X4 Pharmaceuticals was previously known as Arsanis, Inc. before rebranding in 2019.

Senti Biosciences is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing innovative gene and cell therapies using its proprietary synthetic biology platform. Founded in 2016, the company aims to address complex diseases through advanced therapeutic solutions. Senti's technology includes engineered allogeneic chimeric antigen receptor natural killer (CAR-NK) cells, which are designed to target and eliminate cancer cells while minimizing damage to healthy tissue. Among its product candidates are SENTI-202, a Logic Gated CAR-NK cell therapy intended for cancer treatment, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences operates as a resident company at Johnson & Johnson Innovation's JLABS in South San Francisco, collaborating with experts in synthetic biology and computation to advance its therapeutic developments.

Celcuity Inc. is a clinical-stage biotechnology company based in Minneapolis, Minnesota, focused on advancing cancer care through innovative diagnostic and therapeutic solutions. The company's CELsignia diagnostic platform analyzes living tumor cells from patients to identify the specific abnormal cellular processes driving their cancer, enabling personalized treatment options. Celcuity is developing the CELsignia HSF test to diagnose new sub-types of HER2-negative breast cancer, as well as the CELsignia MP test to identify multiple cancer sub-types across various types, including breast, lung, colon, ovarian, kidney, and bladder cancers. In addition to its diagnostic efforts, Celcuity is advancing a lead therapeutic candidate, gedatolisib, a potent pan-PI3K and mTOR inhibitor, which is currently being evaluated in a Phase 3 clinical trial for patients with HR+/HER2- advanced breast cancer. Founded in 2011, Celcuity aims to transform the treatment landscape for patients with cancer by providing targeted therapies tailored to their unique disease profiles.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.

Sorriso Pharmaceuticals is a biotechnology company based in San Diego, California, founded in 2020. The company focuses on developing disease-modifying antibodies aimed at treating inflammatory diseases such as Crohn's disease and ulcerative colitis. Sorriso's innovative platform produces potent antibodies that can be delivered orally, ensuring they remain effective throughout the intestinal system. This approach aims to provide clinical benefits comparable to traditional injectable antibody therapeutics, thereby alleviating the treatment burden for patients who typically rely on injections for their care. By advancing its pipeline of oral therapies, Sorriso Pharmaceuticals seeks to improve patient outcomes and enhance the management of inflammatory diseases.

AviadoBio mission is to transform the lives of people living with neurodegenerative disorders by developing and delivering transformative gene therapies for diseases including frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS). The Company’s technology is based on pioneering research from King’s College London and the UK Dementia Research Institute.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company dedicated to the discovery and development of therapies that target microRNAs to address various diseases. Its primary product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, a severe kidney condition, and RGLS4326, an anti-miR aimed at miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. In addition to these lead candidates, Regulus is cultivating a preclinical pipeline that includes RGLS5579, targeting miR-10b, as well as programs focused on Hepatitis B and Non-Alcoholic Steatohepatitis. Founded in 2007, Regulus Therapeutics is headquartered in San Diego, California.

Recludix pharma is a developing platform to discover potent and selective inhibitors of challenging protein targets. The platform approach integrates custom generated DNA-encoded libraries, massively parallel determination of structure-activity relationships, and a proprietary screening tool to ensure selectivity.

Slope.io, Inc. is a company based in Mobile, Alabama, that specializes in clinical trial logistics for sponsors, contract research organizations (CROs), and research sites across the United States. Established in 2016, Slope offers a platform that effectively connects clinical trials to their supply chains, managing the complexities of delivering devices, drugs, and supply kits to various locations. Their services include Slope.io Coordination, which organizes the logistics requirements of clinical trials into structured timelines and workflows. Additionally, Slope provides customizable supply kits tailored to the specific needs of sites, patients, or study time points, ensuring real-time coordination and complete traceability throughout the process.

858 Therapeutics is a stealth company specializing in the area of healthcare services, pharmaceutical, and biotechnology. The company was founded in 2019 and is headquartered in San Diego, California, USA.

Allay Therapeutics offers analgesic products for post-surgical pain management and recuperation. The company was founded in 2017 and is headquartered in Menlo Park, California, United States.

Stablix Therapeutics is a biotechnology company pioneering the field of Targeted Protein Stabilization (TPS). The company’s resorted platform generates heterobifunctional small molecules (RESTORACS) that recruit deubiquitinase enzymes to remove ubiquitin from targeted proteins and consequently stabilize or increase target protein levels and activity. Stablix initially is leveraging the platform to develop programs to treat rare diseases, cancer, and immunological disorders.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.

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 privately held development-stage company founded in 2015 and located in Montgomery County, Maryland. The Arcellx team is devoted to providing patients with superior immune cell therapies through scientific innovation, accelerated development, and responsible patient care. Although our initial clinical focus is cancer therapy, we are committed to extending our Antigen- Receptor Complex T cell (ARC-T) therapies across a broad spectrum of human disease.

Monte Rosa Therapeutics is a biotechnology company based in Basel, Switzerland, that specializes in developing innovative cancer therapeutics through the modulation of protein degradation pathways. The company focuses on molecular glue degraders (MGDs), a class of small molecule drugs that utilize the body's natural mechanisms for protein degradation to selectively target and degrade proteins relevant to cancer treatment. Monte Rosa employs its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, to identify and develop target proteins for degradation. With a library of over 50,000 MGD molecules, its leading product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing potential for treating MYC-driven tumors. The company is committed to advancing its pipeline of novel therapeutics to address unmet medical needs in oncology.

Satsuma Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative treatments for migraines. The company's primary product candidate, STS101, is a drug-device combination featuring a proprietary dry-powder formulation of dihydroergotamine mesylate. This medication is designed for self-administration using a pre-filled, single-use nasal delivery device. Currently, STS101 is undergoing Phase III clinical trials, marking a significant step in its development process. Founded in 2016 and based in South San Francisco, California, Satsuma Pharmaceuticals aims to provide effective solutions for individuals suffering from acute migraine attacks.

Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.

Personal Genome Diagnostics develops a patient-specific analysis of the cancer genome by using advanced tools and technologies. They were the first to sequence and analyze whole human cancer exomes and subsequently identified many alterations responsible for the development of cancer. They have described the principles underlying the genetic basis of human cancer and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Through PGDx, our founders bring their proprietary methods and expertise to research teams worldwide. The services help oncologists and translational scientists sequence and analyze cancer genomes and identify mutations to characterize aspects of the disease. Their expertise in genome analysis ranges from sample preparation and sequencing to data interpretation and analysis. They specialize in high-throughput next-generation sequencing and proprietary algorithms to identify alterations in complex cancer genomes and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Personal Genome Diagnostics was founded in 2010 and is based in Baltimore, Maryland, United States.

SetPoint Medical Corporation is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices for treating chronic inflammatory diseases, including rheumatoid arthritis and Crohn's Disease. The company leverages the Inflammatory Reflex, a natural mechanism that allows the central nervous system to regulate the immune system, which has been extensively studied and documented in scientific literature. SetPoint's proprietary microregulator aims to enhance this reflex through vagus nerve stimulation, offering an alternative treatment option that provides systemic anti-inflammatory effects. The devices are designed to supplement the body's natural responses and are expected to deliver comparable anti-inflammatory potency to leading medications, while potentially offering improved safety and lower costs. Currently, SetPoint is conducting human trials for its technology at multiple centers in Europe.

Senti Biosciences is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing innovative gene and cell therapies using its proprietary synthetic biology platform. Founded in 2016, the company aims to address complex diseases through advanced therapeutic solutions. Senti's technology includes engineered allogeneic chimeric antigen receptor natural killer (CAR-NK) cells, which are designed to target and eliminate cancer cells while minimizing damage to healthy tissue. Among its product candidates are SENTI-202, a Logic Gated CAR-NK cell therapy intended for cancer treatment, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences operates as a resident company at Johnson & Johnson Innovation's JLABS in South San Francisco, collaborating with experts in synthetic biology and computation to advance its therapeutic developments.

Reneo Pharmaceuticals develops therapies for patients with rare genetic mitochondrial diseases, which impact cellular metabolism and energy production. Founded in 2014 and based in San Diego, California, the company is focused on creating treatments that enhance mitochondrial function, thereby improving muscle health and overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes essential for energy metabolism and the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. By addressing the underlying metabolic deficiencies in these patients, Reneo aims to prevent muscle injury, weakness, and wasting, ultimately enhancing daily functioning for those with these orphan diseases.

Monte Rosa Therapeutics is a biotechnology company based in Basel, Switzerland, that specializes in developing innovative cancer therapeutics through the modulation of protein degradation pathways. The company focuses on molecular glue degraders (MGDs), a class of small molecule drugs that utilize the body's natural mechanisms for protein degradation to selectively target and degrade proteins relevant to cancer treatment. Monte Rosa employs its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, to identify and develop target proteins for degradation. With a library of over 50,000 MGD molecules, its leading product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing potential for treating MYC-driven tumors. The company is committed to advancing its pipeline of novel therapeutics to address unmet medical needs in oncology.

Polyneuron Pharmaceuticals AG is focused on developing innovative therapeutics for the treatment of antibody-mediated autoimmune disorders. The company is working on several drug candidates, including PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron utilizes a platform technology called Antibody-Catch, which is based on biodegradable glycopolymers. This technology selectively targets and eliminates pathological autoantibodies while preserving the integrity of the rest of the immune system. Founded in 2014 and headquartered in Basel, Switzerland, Polyneuron aims to provide safe and effective treatment options for patients suffering from autoimmune diseases affecting the peripheral nervous system.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for autoimmune and neurodegenerative diseases that are influenced by the classical complement pathway. The company’s research revolves around C1q, a key molecule that, when aberrantly activated, contributes to various disease processes. Its lead product candidates include ANX005, a monoclonal antibody designed for intravenous administration, which has completed Phase 1b trials for treating Guillain-Barré syndrome, and ANX007, formulated for intravitreal use, which has also completed Phase 1b trials aimed at glaucoma treatment. Additionally, Annexon is advancing preclinical candidates targeting conditions such as warm autoimmune hemolytic anemia, Huntington's disease, amyotrophic lateral sclerosis, and geographic atrophy. Founded in 2011 and headquartered in South San Francisco, California, the company employs a biomarker-driven strategy to ensure effective engagement of its therapies in the intended patient populations.

Monte Rosa Therapeutics is a biotechnology company based in Basel, Switzerland, that specializes in developing innovative cancer therapeutics through the modulation of protein degradation pathways. The company focuses on molecular glue degraders (MGDs), a class of small molecule drugs that utilize the body's natural mechanisms for protein degradation to selectively target and degrade proteins relevant to cancer treatment. Monte Rosa employs its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, to identify and develop target proteins for degradation. With a library of over 50,000 MGD molecules, its leading product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing potential for treating MYC-driven tumors. The company is committed to advancing its pipeline of novel therapeutics to address unmet medical needs in oncology.

Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating 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 combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.

Affinia Therapeutics Inc. is a biotechnology company focused on developing gene therapies for various diseases, particularly those affecting muscle and the central nervous system (CNS). Founded in 2019 and based in Waltham, Massachusetts, the company specializes in creating rationally designed adeno-associated virus (AAV) vectors. By integrating synthetic and systems biology with high-throughput screening and advanced resolution techniques, Affinia Therapeutics aims to address significant unmet medical needs. The company’s innovative approach allows for the development of novel gene therapies with enhanced properties, targeting the treatment of devastating diseases and improving the lives of affected individuals.

Inventiva S.A. is a biopharmaceutical company based in Daix, France, specializing in the development of oral small molecule therapies targeting significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company's lead candidate, Lanifibranor, is currently in Phase IIb clinical trials for the treatment of nonalcoholic steatohepatitis. Additionally, Inventiva is advancing Odiparcil, in Phase IIa trials for mucopolysaccharidosis type VI, and has several preclinical programs, including potential treatments for malignant mesothelioma and lung cancer. The company's research and development efforts are supported by a proprietary library of molecules and a team experienced in targeting nuclear receptors and transcription factors. Inventiva has established partnerships with prominent organizations, including the Institut Curie for oncology research and AbbVie for autoimmune disease treatments. Founded in 2011, the company continues to focus on innovative solutions for complex medical conditions.

Geron Corporation is a late-stage clinical biopharmaceutical company based in Menlo Park, California, specializing in the development and commercialization of therapeutics for hematologic myeloid malignancies. The company's primary focus is on imetelstat, a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. Imetelstat aims to inhibit the uncontrolled growth of malignant progenitor cells in these cancers, which helps reduce the production of dysfunctional blood cells and facilitates the recovery of normal blood cell production. Founded in 1990, Geron is dedicated to advancing innovative treatments for patients with serious blood disorders.

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.

At Affimed, they are committed to improving outcomes for patients with cancer. Their scientific team has been devoted to unlocking the power of the innate immune system for nearly two decades to provide a unqiue approach to fighting cancer. With their unparalleled expertise in innate immunity and innate cell engager-based medicines, they aim to overcome the limitations and challenges faced by current immuno-oncology (I-O) therapies.

ChromaCode, Inc. is a molecular diagnostics company based in Carlsbad, California, that specializes in developing innovative testing solutions for infectious diseases, cancer, and non-invasive prenatal testing (NIPT) using proprietary technology derived from Caltech. The company enhances the performance of quantitative polymerase chain reaction (qPCR) instruments, achieving a five-fold increase in multiplexing capabilities while maintaining cost-effectiveness. ChromaCode employs advanced bioinformatics techniques and patented mathematical methods to improve signal processing, enabling laboratories to conduct comprehensive testing of multiple targets in a single sample. Additionally, the company offers products such as the HDPCR Tick-Borne Pathogen Panel, which detects various tick-borne pathogens through a multiplex polymerase chain reaction test. Founded in 2012, ChromaCode draws expertise from professionals with backgrounds in leading organizations such as Illumina and Google[x].

Arcellx is a privately held development-stage company founded in 2015 and located in Montgomery County, Maryland. The Arcellx team is devoted to providing patients with superior immune cell therapies through scientific innovation, accelerated development, and responsible patient care. Although our initial clinical focus is cancer therapy, we are committed to extending our Antigen- Receptor Complex T cell (ARC-T) therapies across a broad spectrum of human disease.

Inventiva S.A. is a biopharmaceutical company based in Daix, France, specializing in the development of oral small molecule therapies targeting significant unmet medical needs in fibrosis, lysosomal storage disorders, and oncology. The company's lead candidate, Lanifibranor, is currently in Phase IIb clinical trials for the treatment of nonalcoholic steatohepatitis. Additionally, Inventiva is advancing Odiparcil, in Phase IIa trials for mucopolysaccharidosis type VI, and has several preclinical programs, including potential treatments for malignant mesothelioma and lung cancer. The company's research and development efforts are supported by a proprietary library of molecules and a team experienced in targeting nuclear receptors and transcription factors. Inventiva has established partnerships with prominent organizations, including the Institut Curie for oncology research and AbbVie for autoimmune disease treatments. Founded in 2011, the company continues to focus on innovative solutions for complex medical conditions.

SetPoint Medical Corporation is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices for treating chronic inflammatory diseases, including rheumatoid arthritis and Crohn's Disease. The company leverages the Inflammatory Reflex, a natural mechanism that allows the central nervous system to regulate the immune system, which has been extensively studied and documented in scientific literature. SetPoint's proprietary microregulator aims to enhance this reflex through vagus nerve stimulation, offering an alternative treatment option that provides systemic anti-inflammatory effects. The devices are designed to supplement the body's natural responses and are expected to deliver comparable anti-inflammatory potency to leading medications, while potentially offering improved safety and lower costs. Currently, SetPoint is conducting human trials for its technology at multiple centers in Europe.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cell therapies for cancer treatment. Founded in 2015 and headquartered in South San Francisco, California, Nkarta aims to enhance the efficacy and tolerability of cell therapies, addressing both hematologic and solid tumor malignancies. The company's approach utilizes chimeric antigen receptors on NK cells, enabling them to recognize and attack tumor cells more effectively. Nkarta's lead product candidates include NKX101, designed to harness innate NK cell biology for cancer detection and destruction, and NKX019, targeting the CD19 antigen to treat various B cell malignancies. By leveraging its NK expansion platform and proprietary cell engineering technologies, Nkarta seeks to provide a more potent and accessible treatment option, capitalizing on the natural ability of NK cells to identify and eliminate abnormal cells while minimizing side effects typically associated with other therapies.

Sagimet Biosciences is a biotechnology company that focuses on developing novel therapeutics to treat important diseases such as liver disease NASH and specific cancers, with a focus on targeting dysfunctional metabolic pathways. It was founded in 2007 and headquartered in San Mateo, California.

Reneo Pharmaceuticals develops therapies for patients with rare genetic mitochondrial diseases, which impact cellular metabolism and energy production. Founded in 2014 and based in San Diego, California, the company is focused on creating treatments that enhance mitochondrial function, thereby improving muscle health and overall quality of life for affected individuals. Its lead product candidate, REN001, is an oral medication designed to modulate genes essential for energy metabolism and the production of adenosine triphosphate (ATP), the primary energy source for cellular processes. By addressing the underlying metabolic deficiencies in these patients, Reneo aims to prevent muscle injury, weakness, and wasting, ultimately enhancing daily functioning for those with these orphan diseases.

Polyneuron Pharmaceuticals AG is focused on developing innovative therapeutics for the treatment of antibody-mediated autoimmune disorders. The company is working on several drug candidates, including PN-1007 for anti-MAG neuropathy, PN-1018 for multifocal motor neuropathy, and PN-032 and PN-056 for ABO-incompatible transplantation. Polyneuron utilizes a platform technology called Antibody-Catch, which is based on biodegradable glycopolymers. This technology selectively targets and eliminates pathological autoantibodies while preserving the integrity of the rest of the immune system. Founded in 2014 and headquartered in Basel, Switzerland, Polyneuron aims to provide safe and effective treatment options for patients suffering from autoimmune diseases affecting the peripheral nervous system.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.

Oyster Point Pharma, Inc. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, that specializes in the discovery, development, and commercialization of innovative therapies for ocular surface diseases. Founded in 2015, the company is primarily focused on its lead product candidate, OC-01, a nicotinic acetylcholine receptor agonist currently undergoing Phase III clinical trials for the treatment of dry eye disease. This condition, which affects over 30 million Americans, is characterized by a chronic lack of sufficient tears, leading to discomfort and potential vision problems. OC-01 is developed as a nasal spray and aims to restore tear film homeostasis by activating the trigeminal parasympathetic pathway, thereby stimulating natural tear production. Additionally, the company is exploring the use of OC-01 for neurotrophic keratitis, further expanding its therapeutic potential in ocular health.

Tiburio Therapeutics is a developer of drug compounds intended to treat tumors and endocrine diseases.The company's compounds aid in the treatment of non-functioning pituitary adenoma using a dopamine-somatostatin chimeric molecule that has the potential to shrink or halt tumor growth, providing patients with effective treatment for rare neuroendocrine tumors and endocrine diseases.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.

Allay Therapeutics offers analgesic products for post-surgical pain management and recuperation. The company was founded in 2017 and is headquartered in Menlo Park, California, United States.

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).

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for autoimmune and neurodegenerative diseases that are influenced by the classical complement pathway. The company’s research revolves around C1q, a key molecule that, when aberrantly activated, contributes to various disease processes. Its lead product candidates include ANX005, a monoclonal antibody designed for intravenous administration, which has completed Phase 1b trials for treating Guillain-Barré syndrome, and ANX007, formulated for intravitreal use, which has also completed Phase 1b trials aimed at glaucoma treatment. Additionally, Annexon is advancing preclinical candidates targeting conditions such as warm autoimmune hemolytic anemia, Huntington's disease, amyotrophic lateral sclerosis, and geographic atrophy. Founded in 2011 and headquartered in South San Francisco, California, the company employs a biomarker-driven strategy to ensure effective engagement of its therapies in the intended patient populations.

Mirum Pharmaceuticals is a biopharmaceutical company focused on the development and commercialization of therapies for debilitating liver diseases. It is involved in the development of Maralixibat for the treatment of pediatric patients with progressive familial intrahepatic cholestasis, or PFIC, and Alagille syndrome, or ALGS. The company is also involved in the development of volixibat for the treatment of adult patients with cholestatic liver diseases.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics that aim to enhance the effectiveness of radiotherapy in cancer treatment. The company’s lead product candidate, avasopasem manganese (GC4419), is a small molecule mimetic of superoxide dismutase designed to address radiation-induced severe oral mucositis in patients with head and neck cancer. It is currently undergoing Phase III clinical trials for this indication and Phase IIa trials for treating radiotherapy-induced esophagitis in lung cancer patients. Additionally, Galera is developing a second candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. Founded in 2012 and headquartered in Malvern, Pennsylvania, Galera Therapeutics is dedicated to transforming cancer treatment through advancements in oxygen metabolic pathways.

PhaseBio Pharmaceuticals is a clinical-stage biopharmaceutical company based in Malvern, Pennsylvania, focused on developing innovative therapies for cardiopulmonary diseases. Its lead product candidate, PB2452, is a reversal agent for the antiplatelet drug ticagrelor and is currently undergoing Phase III clinical trials for patients experiencing uncontrolled bleeding or requiring urgent surgery. The company is also advancing PB1046, a fusion protein aimed at treating pulmonary arterial hypertension, which is in Phase IIb clinical trials. Additionally, PhaseBio is developing PB6440, an oral agent intended for the treatment of resistant hypertension. Founded in 2002, the company leverages proprietary recombinant biopolymers to enhance the stability and efficacy of its drug candidates.

Atterocor, Inc. focuses on research and development of therapeutics for adrenal disease. Atterocor, Inc. was incorporated in 2012 and is based in Ann Arbor, Michigan.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.

MEI Pharma, Inc. (Nasdaq: MEIP) is a San Diego-based oncology company focused on the clinical development of novel therapies for cancer. The Company’s clinical development pipeline includes lead drug candidate Pracinostat, a potential best-in-class, oral histone deacetylase (HDAC) inhibitor. Pracinostat has been tested in more than 150 patients in multiple Phase I and exploratory Phase II clinical trials, including advanced hematologic malignancies such as myelodysplastic syndrome, acute myeloid leukemia and myelofibrosis. The Company expects to initiate a randomized Phase II trial of Pracinostat in combination with standard-of-care in at least one hematologic malignancy toward the middle of 2013. In addition, MEI Pharma is developing two drug candidates derived from its isoflavone-based technology platform, ME-143 and ME-344. Results from a Phase I trial of intravenous ME-143 in heavily treated patients with solid refractory tumors were presented at the American Society of Clinical Oncology Annual Meeting in June 2012. A Phase I clinical trial of intravenous ME-344 in patients with solid refractory tumors is ongoing.

Senti Biosciences is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing innovative gene and cell therapies using its proprietary synthetic biology platform. Founded in 2016, the company aims to address complex diseases through advanced therapeutic solutions. Senti's technology includes engineered allogeneic chimeric antigen receptor natural killer (CAR-NK) cells, which are designed to target and eliminate cancer cells while minimizing damage to healthy tissue. Among its product candidates are SENTI-202, a Logic Gated CAR-NK cell therapy intended for cancer treatment, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences operates as a resident company at Johnson & Johnson Innovation's JLABS in South San Francisco, collaborating with experts in synthetic biology and computation to advance its therapeutic developments.

Liquidia Technologies is a late-stage clinical biopharmaceutical company based in Morrisville, North Carolina, that specializes in the development and commercialization of human therapeutics. The company utilizes its proprietary PRINT technology, a particle engineering platform that allows for the precise production of uniform drug particles, aimed at improving the safety, efficacy, and performance of various therapies. Liquidia is currently advancing two key product candidates: LIQ861 for the treatment of pulmonary arterial hypertension and LIQ865 for addressing local post-operative pain. In addition to its internal pipeline, Liquidia collaborates with leading pharmaceutical companies to leverage its PRINT technology across multiple therapeutic areas and drug types. The company also offers strategy and commercialization services for rare disease pharmaceutical products, including a generic formulation of treprostinil for pulmonary arterial hypertension. Founded in 2004, Liquidia Technologies continues to focus on transforming patient care through innovative therapeutic solutions.

Personal Genome Diagnostics develops a patient-specific analysis of the cancer genome by using advanced tools and technologies. They were the first to sequence and analyze whole human cancer exomes and subsequently identified many alterations responsible for the development of cancer. They have described the principles underlying the genetic basis of human cancer and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Through PGDx, our founders bring their proprietary methods and expertise to research teams worldwide. The services help oncologists and translational scientists sequence and analyze cancer genomes and identify mutations to characterize aspects of the disease. Their expertise in genome analysis ranges from sample preparation and sequencing to data interpretation and analysis. They specialize in high-throughput next-generation sequencing and proprietary algorithms to identify alterations in complex cancer genomes and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Personal Genome Diagnostics was founded in 2010 and is based in Baltimore, Maryland, United States.

Centrexion Therapeutics Corporation is a late clinical-stage biopharmaceutical company dedicated to developing novel, non-opioid, and non-addictive therapies for chronic pain management. Founded in 2013 and based in Boston, Massachusetts, the company’s product pipeline includes CNTX-4975, currently undergoing Phase III trials for knee osteoarthritis pain and Phase II trials for Morton’s neuroma and canine osteoarthritis. Additionally, CNTX-0290 is in Phase I trials for various chronic pain conditions, while CNTX-6970 targets inflammatory pain and CNTX-2022, a high-concentration topical gel formulation of lidocaine, is also in Phase I trials for superficial musculoskeletal pain and related conditions. Furthermore, CNTX-6016 is a pre-clinical candidate aimed at treating chronic neuropathic pain. Centrexion Therapeutics is committed to addressing the unmet medical needs in chronic pain treatment by focusing on safety and efficacy.

CTI BioPharma Corp. a biopharmaceutical company, develops, acquires, and commercializes oncology products for cancer treatment. Its development portfolio includes Pixantrone, a phase III trial product, for non-Hodgkin's lymphoma; Brostallicin, which is in first-line Phase II study for the treatment of sarcoma; and OPAXIO, a chemotherapeutic agent for the potential treatment of non-small cell lung, ovarian, and other cancers. It has collaboration and licensing arrangements with Novartis International Pharmaceutical, Ltd.; and PG-TXL Company, L.P. The company was founded in 1991 and is headquartered in Seattle, Washington.

Pionyr Immunotherapeutics Inc. is a biotechnology company based in San Francisco, California, focused on developing innovative cancer immunotherapies. Established in 2015, the company aims to enhance the body’s antitumor immunity by targeting the tumor microenvironment. Pionyr employs advanced technologies for novel target discovery and antibody generation, which underpin its next-generation immuno-oncology therapeutics. Central to its strategy is the Myeloid Tuning™ approach, designed to specifically modify the cellular composition within the tumor microenvironment, thereby improving the immune system's ability to combat tumors.

Allakos Inc. is a clinical-stage biopharmaceutical company based in Redwood City, California, founded in 2012. The company specializes in developing antibody-based therapeutics aimed at treating various diseases linked to dysregulation of the T-helper type 2 immune response, including allergic diseases, inflammation, and conditions characterized by excessive inflammatory cell production. Its lead therapeutic candidate, antolimab (AK002), targets conditions such as eosinophilic gastritis, eosinophilic gastroenteritis, chronic urticaria, indolent systemic mastocytosis, and severe allergic conjunctivitis. Additionally, Allakos is advancing another program, AK006, which targets the Siglec-6 receptor on mast cells to modulate their activity and reduce excessive activation. Through these initiatives, Allakos aims to address significant unmet medical needs in immunological health.

Metacrine, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, dedicated to developing innovative therapies for liver and gastrointestinal diseases. The company is advancing its lead programs, which target the farnesoid X receptor (FXR), a key drug target in these conditions. Currently, MET409 is undergoing a Phase Ib proof-of-concept clinical trial for patients with non-alcoholic steatohepatitis, while MET642 is in a Phase 1 clinical trial for the same patient population. In addition to its FXR-focused programs, Metacrine is exploring a pipeline of novel drug candidates through its internal research and development capabilities. The company also collaborates with Novo Nordisk A/S to further research related to fibroblast growth factor 1. Metacrine was founded in 2014 and is committed to addressing unmet medical needs in liver and gastrointestinal health.

Oyster Point Pharma, Inc. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, that specializes in the discovery, development, and commercialization of innovative therapies for ocular surface diseases. Founded in 2015, the company is primarily focused on its lead product candidate, OC-01, a nicotinic acetylcholine receptor agonist currently undergoing Phase III clinical trials for the treatment of dry eye disease. This condition, which affects over 30 million Americans, is characterized by a chronic lack of sufficient tears, leading to discomfort and potential vision problems. OC-01 is developed as a nasal spray and aims to restore tear film homeostasis by activating the trigeminal parasympathetic pathway, thereby stimulating natural tear production. Additionally, the company is exploring the use of OC-01 for neurotrophic keratitis, further expanding its therapeutic potential in ocular health.

Cydan Development, Inc. is a company focused on the development and commercialization of therapies for orphan and rare diseases, with a particular emphasis on rare genetic disorders. Founded in 2012 and located in Lincoln, Massachusetts, Cydan functions as an orphan drug accelerator, identifying and de-risking compounds with therapeutic potential. The organization conducts comprehensive studies to support the further development of treatments by biotechnology firms. Cydan has worked on therapies for conditions such as Niemann-Pick Disease Type C and IMR-687 for sickle cell disease. The company leverages strong relationships with academic institutions and patient advocacy groups, along with deep expertise in drug development, to foster successful product commercialization and advance treatment options for underserved patient populations.

ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.

SetPoint Medical Corporation is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices for treating chronic inflammatory diseases, including rheumatoid arthritis and Crohn's Disease. The company leverages the Inflammatory Reflex, a natural mechanism that allows the central nervous system to regulate the immune system, which has been extensively studied and documented in scientific literature. SetPoint's proprietary microregulator aims to enhance this reflex through vagus nerve stimulation, offering an alternative treatment option that provides systemic anti-inflammatory effects. The devices are designed to supplement the body's natural responses and are expected to deliver comparable anti-inflammatory potency to leading medications, while potentially offering improved safety and lower costs. Currently, SetPoint is conducting human trials for its technology at multiple centers in Europe.

Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating 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 combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.

Complexa, Inc. is a biopharmaceutical company based in Berwyn, Pennsylvania, focused on developing innovative treatments for severe diseases involving fibrosis and inflammation. Founded in 2008, Complexa specializes in the research and development of endogenous human cell signaling technologies, particularly through the synthesis of endogenous nitro-fatty acids. Their lead compound, CXA-10, is an oral nitrated fatty acid being developed for the treatment of focal segmental glomerulosclerosis and pulmonary arterial hypertension. The company aims to enhance existing anti-inflammatory and metabolic signaling pathways to promote recovery from acute and chronic tissue injuries and diseases, targeting a range of inflammatory, fibrotic, and central nervous system disorders.

Trevi Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in New Haven, Connecticut, dedicated to developing and commercializing nalbuphine ER, an investigational therapy. The company's lead product, Haduvio, is an oral extended-release formulation of nalbuphine currently undergoing Phase IIb/III clinical trials. Trevi Therapeutics is focused on addressing serious neurologically mediated conditions, including chronic pruritus, chronic cough associated with idiopathic pulmonary fibrosis, and levodopa-induced dyskinesia in Parkinson's disease patients. The ongoing PRISM trial evaluates Haduvio's effectiveness in treating severe pruritus related to prurigo nodularis, highlighting its potential in managing conditions linked by a common pathophysiology involving opioid receptors in the nervous system. Founded in 2011, Trevi Therapeutics aims to provide innovative therapeutic solutions for patients with significant unmet medical needs.

Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

OrphoMed, Inc. develops therapies for irritable bowel syndrome with diarrhea (IBS-D), acute and chronic pain, and cystic fibrosis. Its pipeline includes ORP-101, a chemical entity designed to create a stable molecule and to confer partial μ agonist and full κ-antagonist receptor pharmacology; ORP-105, a non-addictive, centrally-acting, non-opiate analgesic; and ORP-110, a metabolically-stable, non-depletory cystic fibrosis transmembrane regulator potentiator. OrphoMed, Inc. was incorporated in 2015 and is based in San Francisco, California.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.

ChromaCode, Inc. is a molecular diagnostics company based in Carlsbad, California, that specializes in developing innovative testing solutions for infectious diseases, cancer, and non-invasive prenatal testing (NIPT) using proprietary technology derived from Caltech. The company enhances the performance of quantitative polymerase chain reaction (qPCR) instruments, achieving a five-fold increase in multiplexing capabilities while maintaining cost-effectiveness. ChromaCode employs advanced bioinformatics techniques and patented mathematical methods to improve signal processing, enabling laboratories to conduct comprehensive testing of multiple targets in a single sample. Additionally, the company offers products such as the HDPCR Tick-Borne Pathogen Panel, which detects various tick-borne pathogens through a multiplex polymerase chain reaction test. Founded in 2012, ChromaCode draws expertise from professionals with backgrounds in leading organizations such as Illumina and Google[x].

SetPoint Medical Corporation is a clinical-stage bioelectronic medicine company based in Valencia, California, focused on developing implantable neuromodulation devices for treating chronic inflammatory diseases, including rheumatoid arthritis and Crohn's Disease. The company leverages the Inflammatory Reflex, a natural mechanism that allows the central nervous system to regulate the immune system, which has been extensively studied and documented in scientific literature. SetPoint's proprietary microregulator aims to enhance this reflex through vagus nerve stimulation, offering an alternative treatment option that provides systemic anti-inflammatory effects. The devices are designed to supplement the body's natural responses and are expected to deliver comparable anti-inflammatory potency to leading medications, while potentially offering improved safety and lower costs. Currently, SetPoint is conducting human trials for its technology at multiple centers in Europe.

Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to the discovery and development of innovative small-molecule pharmaceutical products aimed at treating central nervous system (CNS) disorders. The company specializes in creating oral allosteric modulators that target G-protein coupled receptors, which are crucial for therapeutic interventions. Its leading programs include Dipraglurant, designed for treating levodopa-induced dyskinesia and dystonia in Parkinson’s disease, ADX71149 for epilepsy and other undisclosed CNS disorders, and a GABAB PAM aimed at addressing addiction. Addex Therapeutics has established a collaboration with Janssen Pharmaceuticals to advance the development of mGluR2PAM compounds for human health applications. Founded in 2002, the company has evolved its focus and pipeline based on a pioneering drug discovery platform that emphasizes allosteric modulation.

Rhythm Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing peptide therapeutics for the treatment of rare genetic disorders, particularly those leading to life-threatening metabolic conditions. Its primary product candidate, setmelanotide, is a first-in-class melanocortin-4 receptor agonist currently undergoing Phase III clinical trials for obesity related to pro-opiomelanocortin and leptin receptor deficiencies, as well as Bardet-Biedl and Alström syndromes. Additionally, setmelanotide is in Phase II trials for various other genetic obesity disorders. The company is also advancing RM-853, an orally available ghrelin O-acyltransferase inhibitor, in preclinical development for Prader-Willi syndrome. Founded in 2008 and headquartered in Boston, Massachusetts, Rhythm Pharmaceuticals aims to address significant unmet medical needs in the realm of genetic metabolic disorders through innovative therapies.

Tarveda Therapeutics is focused on developing innovative cancer therapies specifically for solid tumors. The company specializes in miniature drug conjugates, with its lead candidate, PEN-866, targeting activated Heat Shock Protein 90 (HSP90) to deliver a potent topoisomerase 1 inhibitor, SN38, directly to tumor cells. Additionally, Tarveda is advancing another drug conjugate aimed at treating patients with neuroendocrine tumors and small cell lung cancer that express somatostatin receptor 2 (SSTR2). By leveraging its targeted drug delivery platform, Tarveda Therapeutics aims to improve treatment outcomes for patients with difficult-to-treat cancers while reducing potential side effects. Founded in 2011 and based in Watertown, Massachusetts, the company was previously known as Blend Therapeutics until its rebranding in 2016.

Regulus Therapeutics Inc. is a clinical-stage biopharmaceutical company dedicated to the discovery and development of therapies that target microRNAs to address various diseases. Its primary product candidates include RG-012, an anti-miR targeting miR-21, currently in Phase II clinical trials for Alport syndrome, a severe kidney condition, and RGLS4326, an anti-miR aimed at miR-17, which is undergoing Phase Ib clinical trials for autosomal dominant polycystic kidney disease. In addition to these lead candidates, Regulus is cultivating a preclinical pipeline that includes RGLS5579, targeting miR-10b, as well as programs focused on Hepatitis B and Non-Alcoholic Steatohepatitis. Founded in 2007, Regulus Therapeutics is headquartered in San Diego, California.

Addex Therapeutics Ltd is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to the discovery and development of innovative small-molecule pharmaceutical products aimed at treating central nervous system (CNS) disorders. The company specializes in creating oral allosteric modulators that target G-protein coupled receptors, which are crucial for therapeutic interventions. Its leading programs include Dipraglurant, designed for treating levodopa-induced dyskinesia and dystonia in Parkinson’s disease, ADX71149 for epilepsy and other undisclosed CNS disorders, and a GABAB PAM aimed at addressing addiction. Addex Therapeutics has established a collaboration with Janssen Pharmaceuticals to advance the development of mGluR2PAM compounds for human health applications. Founded in 2002, the company has evolved its focus and pipeline based on a pioneering drug discovery platform that emphasizes allosteric modulation.

Immunomedics, Inc. is a clinical-stage biopharmaceutical company based in Morris Plains, New Jersey, that focuses on developing monoclonal antibody-based therapies for cancer treatment. The company's lead product, sacituzumab govitecan, is an antibody-drug conjugate targeting metastatic triple-negative breast cancer, where it is positioned as a third-line treatment in the United States. Additionally, Immunomedics is advancing several other candidates, including IMMU-140 for hematologic malignancies and IMMU-130 for colorectal cancer. The company also explores collaborative clinical efforts with major pharmaceutical partners to enhance the development and application of its products. Founded in 1982, Immunomedics aims to deliver innovative treatments to patients with serious diseases, leveraging its capabilities to drive scientific advancements and operational effectiveness. As of late 2020, it operates as a subsidiary of Gilead Sciences, Inc.

Trillium Therapeutics Inc. is a clinical-stage immuno-oncology company based in Mississauga, Canada, focused on developing therapies for cancer treatment. Its leading program, TTI-621, is a SIRPaFc fusion protein designed to inhibit the CD47 signal, currently undergoing Phase I clinical trials for advanced relapsed or refractory hematologic malignancies, solid tumors, and mycosis fungoides. The company is also advancing TTI-622, another SIRPaFc protein in Phase I trials, and TTI-10001, a small molecule stimulator of interferon genes agonist in the discovery phase aimed at enhancing tumor immunity. Trillium Therapeutics, originally founded in 2004 and previously known as Stem Cell Therapeutics Corp., specializes in the discovery and development of protein therapeutics, including monoclonal antibodies and peptides, with a focus on cancer and other therapeutic areas.

Cleave Therapeutics, Inc. is a biopharmaceutical company based in San Francisco, California, founded in 2010. The company specializes in developing novel small-molecule drugs that target valosin-containing protein and other protein-degradation pathways critical for the survival of cancer cells. By focusing on these mechanisms, Cleave Therapeutics aims to create enzyme inhibitors that can effectively treat cancer and neurodegenerative diseases. Its research efforts are directed toward discovering innovative therapies that may improve patient outcomes in oncology.

Vtesse, Inc. develops drugs for patients suffering from rare diseases. The company focuses on conducting the clinical study of VTS-270 for the treatment of Niemann-Pick disease type C (NPC), as well as on conducting pre-clinical discovery and development of other novel drugs for NPC and other lysosomal storage diseases. Vtesse, Inc. is based in Gaithersburg, Maryland. As of April 3, 2017, Vtesse, Inc. operates as a subsidiary of Sucampo Pharmaceuticals, Inc.

Ardelyx, Inc. is a biopharmaceutical company focused on developing and commercializing innovative therapies for cardiorenal diseases. Headquartered in Fremont, California, Ardelyx's lead product candidate, tenapanor, has successfully completed Phase 3 clinical trials for treating irritable bowel syndrome with constipation and is also in Phase 3 trials for managing hyperphosphatemia in end-stage renal disease patients undergoing dialysis. The company is advancing RDX013, a small molecule potassium secretagogue, aimed at treating hyperkalemia. Ardelyx is also developing RDX5791, an oral NHE3 sodium transport inhibitor targeting constipation-predominant irritable bowel syndrome and excess dietary sodium absorption, currently in Phase 2 trials. Additionally, RDX002 and RDX009, which inhibit phosphate absorption and target type 2 diabetes, respectively, are in preclinical development. Ardelyx emphasizes non-systemic oral therapeutics that minimize side effects while effectively addressing metabolic disorders. Since its founding in 2007, the company has secured significant funding to support its research and development efforts.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing innovative therapies for autoimmune and neurodegenerative diseases that are influenced by the classical complement pathway. The company’s research revolves around C1q, a key molecule that, when aberrantly activated, contributes to various disease processes. Its lead product candidates include ANX005, a monoclonal antibody designed for intravenous administration, which has completed Phase 1b trials for treating Guillain-Barré syndrome, and ANX007, formulated for intravitreal use, which has also completed Phase 1b trials aimed at glaucoma treatment. Additionally, Annexon is advancing preclinical candidates targeting conditions such as warm autoimmune hemolytic anemia, Huntington's disease, amyotrophic lateral sclerosis, and geographic atrophy. Founded in 2011 and headquartered in South San Francisco, California, the company employs a biomarker-driven strategy to ensure effective engagement of its therapies in the intended patient populations.

Verona Pharma is a clinical-stage biopharmaceutical company headquartered in London, United Kingdom, that specializes in developing therapies for chronic respiratory diseases with significant unmet medical needs. The company's lead product candidate, ensifentrine, is an inhaled dual inhibitor of phosphodiesterase 3 and 4, functioning as both a bronchodilator and an anti-inflammatory agent. Ensifentrine is currently in Phase 2b clinical development as a nebulized formulation for the maintenance treatment of chronic obstructive pulmonary disease (COPD), and alternative delivery methods, including a dry powder inhaler and a pressurized metered-dose inhaler, are also being explored. In addition to COPD, Verona Pharma aims to develop ensifentrine for other respiratory conditions such as cystic fibrosis and asthma. Founded in 2005, the company is committed to improving the health and quality of life for individuals affected by these challenging diseases.

AVEO Oncology is a biopharmaceutical company engaged in the development and commercialization of targeted therapies for oncology and other unmet medical needs. The company's lead product, tivozanib, is an oral, once-daily inhibitor of vascular endothelial growth factor receptors, primarily indicated for renal cell carcinoma (RCC). AVEO has completed a Phase III trial (TIVO-3) for tivozanib and is conducting a Phase Ib/II trial combining it with Opdivo, an immune checkpoint inhibitor. Additionally, AVEO is developing Ficlatuzumab, an antibody currently in Phase II trials targeting various cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company's preclinical pipeline includes AV-380, aimed at cachexia, and AV-353, for pulmonary arterial hypertension. AVEO has established multiple collaboration agreements with various pharmaceutical companies and is involved in clinical trials assessing IMFINZI, an antibody targeting PD-L1. Headquartered in Boston, Massachusetts, AVEO was incorporated in 2001 and has a focus on advancing cancer therapeutics through its proprietary cancer biology platform.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.

TESARO, Inc. is an oncology-focused biopharmaceutical company established in 2010 and based in Waltham, Massachusetts. The company specializes in the identification, acquisition, development, and commercialization of cancer therapeutics and supportive care products. Its notable offerings include ZEJULA, an oral poly polymerase inhibitor used for the maintenance treatment of recurrent ovarian cancer, and VARUBI, a neurokinin-1 receptor antagonist designed to prevent chemotherapy-induced nausea and vomiting. TESARO is also advancing a range of immunotherapy candidates, such as TSR-042, TSR-022, and TSR-033, all currently in phase I clinical trials. The company has formed various collaboration and licensing agreements with industry partners, including AnaptysBio, Janssen Biotech, and Genentech. In January 2019, TESARO became a subsidiary of GlaxoSmithKline plc, further enhancing its capabilities in cancer treatment innovation.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the discovery and development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is an oral therapy currently in Phase I/IIa clinical trials for the treatment of phenylketonuria. Additionally, Synlogic is developing SYNB1891, an intratumoral synthetic biotic medicine that is undergoing Phase I clinical trials for the treatment of solid tumors and lymphoma. The company is also exploring synthetic biotics for more common conditions, including inflammatory bowel disease and secondary hyperoxaluria. Synlogic has established a collaboration with Ginkgo Bioworks to advance its development of these innovative therapies.

Galera Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing novel therapeutics that aim to enhance the effectiveness of radiotherapy in cancer treatment. The company’s lead product candidate, avasopasem manganese (GC4419), is a small molecule mimetic of superoxide dismutase designed to address radiation-induced severe oral mucositis in patients with head and neck cancer. It is currently undergoing Phase III clinical trials for this indication and Phase IIa trials for treating radiotherapy-induced esophagitis in lung cancer patients. Additionally, Galera is developing a second candidate, GC4711, which is in Phase I/II trials for pancreatic and lung cancers. Founded in 2012 and headquartered in Malvern, Pennsylvania, Galera Therapeutics is dedicated to transforming cancer treatment through advancements in oxygen metabolic pathways.

Tarveda Therapeutics is focused on developing innovative cancer therapies specifically for solid tumors. The company specializes in miniature drug conjugates, with its lead candidate, PEN-866, targeting activated Heat Shock Protein 90 (HSP90) to deliver a potent topoisomerase 1 inhibitor, SN38, directly to tumor cells. Additionally, Tarveda is advancing another drug conjugate aimed at treating patients with neuroendocrine tumors and small cell lung cancer that express somatostatin receptor 2 (SSTR2). By leveraging its targeted drug delivery platform, Tarveda Therapeutics aims to improve treatment outcomes for patients with difficult-to-treat cancers while reducing potential side effects. Founded in 2011 and based in Watertown, Massachusetts, the company was previously known as Blend Therapeutics until its rebranding in 2016.

Atterocor, Inc. focuses on research and development of therapeutics for adrenal disease. Atterocor, Inc. was incorporated in 2012 and is based in Ann Arbor, Michigan.

Cascadian Therapeutics, a clinical-stage biopharmaceutical company, researches and develops, and sells therapeutic products for the treatment of cancer in the United States. The company lead clinical-stage product candidate is tucatinib, an orally active and HER2-selective small molecule tyrosine kinase inhibitor, which is in two Phase Ib trials, one in combination with Kadcyla and another in combination with Xeloda and/or Herceptin. It is also developing Checkpoint kinase 1, a protein kinase that is in pre-clinical studies to regulate the cell division cycle, as well as to DNA damage and replication stress. In addition, the company focuses on the development of novel antibodies and T-cell immunoreceptor with Ig and ITM domains, an inhibitory receptor expressed on T-cells and NK cells that negatively regulate immune response to cancers. It has a license agreement with Array BioPharma Inc. to develop, manufacture and commercialize tucatinib; research collaboration agreement with Sentinel Oncology Ltd. for the discovery of novel Chk1 inhibitors; and collaboration agreement with Adimab LLC for the discovery of novel antibodies against d immunotherapy targets in oncology.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing innovative therapeutics for patients with rare genetic disorders of hemoglobin, particularly sickle cell disease and beta-thalassemia. Founded in 2016, the company is advancing IMR-687, a once-daily oral therapy designed to be a potent small molecule inhibitor of PDE9. This therapeutic aims to address the significant health challenges associated with these conditions, which can lead to reduced healthy red blood cells and various complications. Imara was established following a collaboration between Cydan Development and H. Lundbeck A/S, with initial funding from notable life science investors.

AVEO Oncology is a biopharmaceutical company engaged in the development and commercialization of targeted therapies for oncology and other unmet medical needs. The company's lead product, tivozanib, is an oral, once-daily inhibitor of vascular endothelial growth factor receptors, primarily indicated for renal cell carcinoma (RCC). AVEO has completed a Phase III trial (TIVO-3) for tivozanib and is conducting a Phase Ib/II trial combining it with Opdivo, an immune checkpoint inhibitor. Additionally, AVEO is developing Ficlatuzumab, an antibody currently in Phase II trials targeting various cancers, and AV-203, an anti-ErbB3 monoclonal antibody that has completed Phase I trials for esophageal cancer. The company's preclinical pipeline includes AV-380, aimed at cachexia, and AV-353, for pulmonary arterial hypertension. AVEO has established multiple collaboration agreements with various pharmaceutical companies and is involved in clinical trials assessing IMFINZI, an antibody targeting PD-L1. Headquartered in Boston, Massachusetts, AVEO was incorporated in 2001 and has a focus on advancing cancer therapeutics through its proprietary cancer biology platform.

ObsEva SA is a clinical-stage biopharmaceutical company based in Geneva, Switzerland, dedicated to developing innovative therapeutics for women's reproductive health and pregnancy. Founded in 2012 by Ernest Loumaye and André Chollet, the company focuses on addressing serious conditions such as endometriosis, heavy menstrual bleeding, and preterm labor. Its key products include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and uterine fibroids, and OBE022, a selective prostaglandin F2α receptor antagonist designed for once-daily treatment of preterm labor. Additionally, ObsEva is advancing Nolasiban, an oral oxytocin receptor antagonist intended to enhance clinical pregnancy and live birth rates in women undergoing in vitro fertilization. The company operates primarily in Switzerland and is committed to improving outcomes for women facing reproductive health challenges.

Amplyx Pharmaceuticals is a small molecule drug development company focused on creating innovative therapies for life-threatening infections, particularly in patients with compromised immune systems. Founded in 2006 and based in San Diego, California, the company specializes in developing oral and injectable anti-infective drugs, including antifungal agents aimed at treating 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 combining them with additional small molecules. This strategy addresses critical treatment needs in hospital settings and provides effective therapy options for patients after discharge, particularly those undergoing chemotherapy and other immunosuppressive treatments.

Nightstar Therapeutics plc is a clinical-stage gene therapy company based in London, focused on developing innovative one-time treatments for rare inherited retinal diseases. The company’s lead product candidate, NSR-REP1, is currently in phase 3 clinical development for choroideremia, while NSR-RPGR is undergoing phase 2/3 trials for X-linked retinitis pigmentosa. Additionally, Nightstar is developing NSR-BEST1 and NSR-ABCA4, with the latter in preclinical development for Stargardt disease. Founded in 2017, Nightstar Therapeutics aims to address the unmet medical needs of patients suffering from these conditions.

Personal Genome Diagnostics develops a patient-specific analysis of the cancer genome by using advanced tools and technologies. They were the first to sequence and analyze whole human cancer exomes and subsequently identified many alterations responsible for the development of cancer. They have described the principles underlying the genetic basis of human cancer and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Through PGDx, our founders bring their proprietary methods and expertise to research teams worldwide. The services help oncologists and translational scientists sequence and analyze cancer genomes and identify mutations to characterize aspects of the disease. Their expertise in genome analysis ranges from sample preparation and sequencing to data interpretation and analysis. They specialize in high-throughput next-generation sequencing and proprietary algorithms to identify alterations in complex cancer genomes and have developed novel technologies for non-invasive “liquid biopsy” approaches in cancer. Personal Genome Diagnostics was founded in 2010 and is based in Baltimore, Maryland, United States.