OrbiMed

Jade Biosciences is developing transformative therapies to redefine the standard of care for inflammation and immunology indications.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

Aadi Bioscience, Inc. is a clinical-stage biopharmaceutical company based in Pacific Palisades, California, focused on developing precision therapies for genetically-defined cancers. The company specializes in mTOR inhibitors, particularly ABI-009, aimed at treating patients with alterations in the TSC1 or TSC2 genes, where existing mTOR inhibitors have limitations in pharmacology, drug delivery, or safety. Aadi's primary focus is on providing transformational therapies for patients with ultra-rare cancers, such as perivascular epithelioid cell tumors (PEComa). Since its founding in 2011, Aadi Bioscience has aimed to address significant unmet medical needs in oncology and related therapeutic areas.

Pulnovo Medical, founded in 2013 in Wuxi, Anhui, specializes in the development, production, and service of innovative medical devices aimed at treating pulmonary hypertension. The company focuses on advanced pulse radiofrequency ablation technology, which includes pioneering high-frequency ablation equipment and intravascular catheters. These products are designed to enhance the quality of life for patients suffering from pulmonary hypertension and heart failure, offering a novel approach to treatment with a commitment to improving patient outcomes. Pulnovo Medical holds global invention patents for its unique technologies, underscoring its role as a leader in this specialized medical field.

Helicore Biopharma is at the forefront of developing cutting-edge treatments for obesity and related disorders.

RAPT Therapeutics is a clinical-stage biopharmaceutical company specializing in immunology. It focuses on discovering, developing, and commercializing oral small-molecule therapies aimed at addressing significant unmet medical needs in oncology and inflammatory diseases. RAPT's proprietary discovery engine has led to the advancement of two drug candidates: FLX475, which selectively inhibits the migration of immunosuppressive regulatory T cells into tumors, and RPT193, which targets type 2 T helper cells in inflamed tissues. Both candidates have progressed to clinical trials within a short time frame since the company's inception. Additionally, RAPT is exploring further therapeutic targets, including general control nonderepressible 2 and hematopoietic progenitor kinase 1, which are currently in the discovery phase.

Aadi Bioscience, Inc. is a clinical-stage biopharmaceutical company based in Pacific Palisades, California, focused on developing precision therapies for genetically-defined cancers. The company specializes in mTOR inhibitors, particularly ABI-009, aimed at treating patients with alterations in the TSC1 or TSC2 genes, where existing mTOR inhibitors have limitations in pharmacology, drug delivery, or safety. Aadi's primary focus is on providing transformational therapies for patients with ultra-rare cancers, such as perivascular epithelioid cell tumors (PEComa). Since its founding in 2011, Aadi Bioscience has aimed to address significant unmet medical needs in oncology and related therapeutic areas.

Angitia Biopharmaceuticals is a global biotechnology company dedicated to discovering and developing innovative therapeutics for serious musculoskeletal diseases. The company focuses on researching and creating breakthrough therapies aimed at treating conditions affecting bones, muscles, and joints. By prioritizing the development of novel drugs, Angitia Biopharmaceuticals aims to improve patient outcomes and facilitate recovery from severe musculoskeletal ailments.

ADCendo ApS is a biotechnology company focused on developing innovative anti-cancer drugs through the use of antibody-drug conjugates (ADCs). The company targets novel receptors that are overexpressed in various cancer types, addressing significant unmet medical needs in the field of oncology. Its ADCs aim to provide targeted therapy for patients suffering from aggressive cancers such as soft-tissue sarcoma, osteosarcoma, glioblastoma multiforme, and triple-negative breast cancer, as well as other carcinomas including those of the breast, colon, pancreas, prostate, ovary, and kidney. Through its research and development efforts, ADCendo strives to create effective treatment options for patients facing challenging cancer diagnoses.

enGene is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.

Chemomab Ltd. is a clinical-stage biotechnology company based in Tel Aviv, Israel, specializing in the development of therapeutic antibodies for autoimmune and inflammatory diseases. The company focuses on discovering innovative treatments for fibrosis-related diseases that have significant unmet medical needs. Chemomab's lead product, CM-101, is a monoclonal antibody designed to inhibit the activity of CCL24, a soluble protein that plays a crucial role in promoting fibrosis and inflammation. CM-101 is currently under clinical development, with a particular emphasis on treating orphan diseases such as Primary Sclerosing Cholangitis and Systemic Sclerosis. The company is conducting three Phase 2 clinical trials for CM-101 in various fibrotic indications and anticipates reporting results from these studies in the near future.

Asceneuron develops effective therapeutics for orphan tauopathies and Alzheimer’s disease. By focusing on areas of high unmet medical need, Asceneuron aspires to become a leading biotech company specialized in small molecule drug discovery for neurodegenerative diseases. Tauopathies are currently untreatable neurodegenerative diseases that rapidly progress towards debilitating conditions. The appearance of deposits of the microtubule-associated tau protein as e.g. neurofibrillary tangles in neurons of the brain is a common feature of tauopathies that is shared with Alzheimer’s disease. Neuronal tau deposits are known to be a major contributor to neurodegeneration and mutations in the tau gene are causative of the tauopathy fronto-temporal dementia and parkinsonism linked to chromosome 17 (FTDP-17). Due to increasing life expectancy, Alzheimer’s disease is viewed as one of the largest healthcare problems of this century, imposing a major economic burden on societies in the Western and developing world. Current treatment options provide limited benefits supporting the urgent need for more efficacious and better tolerated medicines that address symptomatic relief as well as disease progression.

Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company based in Burlingame, California, dedicated to developing innovative immuno-oncology therapies. The company’s lead product candidate, Ciforadenant (CPI-444), is an oral small molecule antagonist of the A2A receptor and is currently undergoing Phase Ib/2 clinical trials. Additionally, Corvus is advancing CPI-006, an anti-CD73 monoclonal antibody, which is in Phase I/Ib trials aimed at inhibiting adenosine production and stimulating immune responses, as well as investigating its potential for treating COVID-19. Another candidate, CPI-818, is a small molecule that selectively inhibits interleukin-2-inducible T-cell kinase and is also in Phase I/Ib trials. Corvus Pharmaceuticals collaborates strategically with Angel Pharmaceuticals to enhance its pipeline of targeted investigational medicines. Founded in 2014, the company is focused on addressing significant unmet medical needs in cancer treatment and related diseases.

TriSalus Life Sciences, based in Westminster, Colorado, focuses on developing innovative drug delivery technologies and immune-oncology therapeutics aimed at improving treatment outcomes for challenging cancers, particularly liver and pancreatic cancer. Founded in 2009, the company specializes in precision infusion systems designed for interventional radiology. Its product offerings include Pressure Enabled Drug Delivery (PEDD) systems, which utilize a unique expandable tip catheter to deliver embolic agents while minimizing damage to healthy tissues. The Surefire infusion systems, including various models for lobar, segmental, and super-selective infusions, aim to enhance tumor uptake through controlled pressure and blood flow management. Additionally, TriSalus is advancing an investigational therapy, nelitolimod, which has shown promise in enhancing immune response against liver tumors. The company continues to innovate with devices like the TriNav, which employs SmartValve technology to optimize drug delivery.

Sparrow Pharmaceuticals is a clinical-stage biopharmaceutical company focused on developing innovative treatments for disorders related to corticosteroid excess. Founded in 2013 by David A. Katz and based in Portland, Oregon, the company aims to provide better therapeutic options for patients suffering from hypercortisolism and autoimmune or inflammatory conditions. By utilizing unique insights into corticosteroid biology, Sparrow is advancing its lead product, SPI-62, an oral small molecule treatment that specifically targets the source of active intracellular corticosteroids in critical tissues. This approach is designed to alleviate the adverse effects of both naturally occurring corticosteroids, such as cortisol due to tumors, and the long-term use of corticosteroid medications like prednisone.

Cullinan Therapeutics is a developer of an externally sourced cancer therapeutics intended to end a drug program quickly if the early research suggests it won't work. The company's therapeutics are developed via assets sourced internally through dry lab or externally through business, academic and pharma collaborations, enabling researchers to get a highly diversified portfolio of oncology therapeutics with a unique, cost-efficient business model.

Alterome Therapeutics is a biotechnology company focused on precision oncology, dedicated to developing targeted therapeutics that specifically address validated oncogenic drivers in cancer. The firm aims to discover and create alteration-specific therapies that provide hope and improved treatment options for individuals affected by cancer. By concentrating on high-value oncogenic alterations, Alterome Therapeutics seeks to enhance the effectiveness of cancer treatments and support healthcare providers in their efforts to combat the disease.

Avalo Therapeutics is a clinical-stage biopharmaceutical company that employs a precision medicine approach to discover, develop, and commercialize highly targeted therapeutics in areas of significant unmet clinical need.

Nkarta Therapeutics is a biopharmaceutical company focused on developing engineered natural killer (NK) cells for the treatment of cancer and autoimmune diseases. The company aims to enhance the efficacy of cell therapy by leveraging the inherent ability of NK cells to identify and eliminate abnormal cells without the need for genetic alteration. This approach allows for a broader application across various hematologic and solid tumor malignancies while potentially minimizing side effects associated with traditional T-cell therapies. Nkarta combines its NK expansion platform technology with proprietary cell engineering methods to produce a substantial supply of NK cells, enhancing their ability to recognize therapeutic targets and improving their persistence in the body for sustained activity. Through this innovative strategy, Nkarta seeks to provide more potent, well-tolerated, and rapidly available therapies for patients facing diverse cancer types.

Q32 Bio Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapies for severe autoimmune and inflammatory diseases. Established in 2017, the company aims to address immune system dysregulation, providing treatment options for conditions that currently have limited or inadequate therapies. Q32 Bio's pipeline includes ADX-914, a human anti-IL-7R antibody designed to re-regulate adaptive immune function, and ADX-097, which demonstrates effective distribution to affected tissues and robust efficacy. With a team of experienced scientists and industry professionals, Q32 Bio is dedicated to restoring healthy immune balance through novel biologic therapies that target critical pathways in both the innate and adaptive immune systems.

Mirador Therapeutics envisions precision medicine for immune-mediated inflammatory and fibrotic diseases.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

Fate Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing programmed cellular immunotherapies for cancer and immune disorders. The company’s pipeline includes various NK- and T-cell immuno-oncology programs, such as FT516 for acute myeloid leukemia and B-cell lymphoma, FT596 for B-cell lymphoma and chronic lymphocytic leukemia, and FT538 for multiple myeloma. Additional programs include FT500, FT576, FT819, and FT-ONO1, targeting solid tumors and hematologic malignancies. Fate Therapeutics is also collaborating with Ono Pharmaceutical Co. Ltd. and Janssen Biotech, Inc. to advance its off-the-shelf iPSC-derived CAR T-cell therapies and other innovative treatments. Founded in 2007, the company leverages insights from leading scientists to create and modulate stem cells for therapeutic applications, aiming to restore health through advanced cellular therapies.

Geron Corporation is a late-stage clinical biopharmaceutical company headquartered in Menlo Park, California, that specializes in developing therapies for hematologic myeloid malignancies. The company's lead product, imetelstat, is a first-in-class telomerase inhibitor currently undergoing Phase 2/3 clinical trials. This investigational drug aims to inhibit the uncontrolled proliferation of malignant progenitor cells, which can lead to dysfunctional blood cell production, thereby facilitating the recovery of normal blood cell development. Founded in 1990, Geron focuses on advancing treatments for conditions such as myelodysplastic syndromes and myelofibrosis, a rare blood cancer affecting bone marrow. The company also generates revenue through collaboration agreements, milestones, royalties, and licensing arrangements.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

ProfoundBio is a clinical-stage biotechnology company dedicated to creating innovative antibody-based therapeutics aimed at treating cancer. Utilizing proprietary technology platforms, the company has developed a diverse pipeline of antibody-drug conjugate (ADC) candidates targeting both solid tumors and hematological malignancies. These drug candidates are currently in various stages of discovery, preclinical, and clinical development. ProfoundBio's focus is on harnessing the immune system to provide patients with more effective treatments that have the potential for curative outcomes.

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

Mineralys Therapeutics is a biopharmaceutical company that provides hypertension treatment. They also bring a targeted approach to the management of hypertension.

Tyra Biosciences is a clinical-stage biotechnology company based in Carlsbad, California, that specializes in developing small molecule therapies for cancer treatment. Founded in 2018, the company aims to address drug resistance in cancer therapies through its proprietary SNÅP platform, which facilitates rapid and precise drug design by generating iterative molecular snapshots. Tyra is particularly focused on creating selective inhibitors of the Fibroblast Growth Factor Receptor (FGFR) family, which are implicated in approximately 7% of all cancers. The company's lead product candidate, TYRA-300, specifically targets FGFR3 and is initially aimed at patients with metastatic urothelial carcinoma of the bladder and urinary tract. Through its innovative approach, Tyra Biosciences seeks to improve treatment outcomes for cancer patients by providing alternatives to existing therapies.

OnCusp Therapeutics is a biopharmaceutical company turning cutting-edge research assets into innovative oncology treatments. It is always working to improve the largest value inflection point in the drug development value chain, believing that speeding oncology medication innovation is the most effective method to provide help and hope to cancer patients around the world. It is founded by Bing Yuan, Eric Slosberg, and Andy Fu.

ENYO Pharma SA is a clinical-stage biopharmaceutical company based in Lyon, France, that focuses on the discovery and development of innovative treatments for acute and chronic viral infections. Established in 2014 by a team of scientists from the Infectiology Research Center, the company utilizes a proprietary platform to identify intracellular therapeutic targets and develop small molecule therapeutics. ENYO Pharma's research primarily targets significant human infecting viruses, with current programs aimed at treating chronic hepatitis B and severe influenza. The company's pipeline includes a lead compound, EYP001, which modulates FXR to reduce the cccDNA reservoir and inhibit the expression of viral proteins, along with EYP002, which is in preclinical studies. With an emphasis on addressing unmet medical needs in infectious diseases and metabolic disorders, ENYO Pharma has established collaborations with prominent research institutions to enhance its drug development efforts.

BlossomHill Therapeutics, Inc. is a small molecule drug discovery and development company focused on unmet medical needs in oncology and autoimmune disorders.

Koya Medical, Inc., founded in 2018 and based in Oakland, California, specializes in developing innovative therapeutic devices aimed at treating cancer-related chronic lymphedema and various venous diseases. The company focuses on creating wearable technology that delivers active compression therapy, which is designed to enhance movement and mobility for patients. Koya Medical's approach offers a personalized alternative to traditional compression methods, utilizing a sensor-based closed-loop system to provide tailored compression patterns. This technology addresses the needs of patients suffering from conditions such as lymphedema, chronic edema, and chronic venous insufficiency, thereby improving their quality of care and overall treatment outcomes.

Terremoto Biosciences is a biotechnology company leveraging the power of lysine-based covalency to develop optimized medicines.

Angitia Biopharmaceuticals is a global biotechnology company dedicated to discovering and developing innovative therapeutics for serious musculoskeletal diseases. The company focuses on researching and creating breakthrough therapies aimed at treating conditions affecting bones, muscles, and joints. By prioritizing the development of novel drugs, Angitia Biopharmaceuticals aims to improve patient outcomes and facilitate recovery from severe musculoskeletal ailments.

Minervax is focused on developing a vaccine to prevent infections caused by Group B streptococcus (GBS), which can lead to serious health issues in newborns and adverse pregnancy outcomes. The company is researching a specific fusion protein that has demonstrated the ability to stimulate strong protective immunity against various clinically significant GBS strains. By utilizing this innovative approach, Minervax aims to provide medical professionals with an effective tool to protect newborns from potentially life-threatening bacterial infections associated with GBS.

Sirius Therapeutics is an innovative biotechnology company that focuses on RNAi therapies for cardiovascular diseases.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Corteria Pharmaceuticals develops first-in-class drugs in heart failure subpopulations, focused on the development of transformative therapies for the treatment of worsening and acute decompensated heart failure.

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

Epigenic Therapeutics is a biotech firm that offers gene editing therapy.

Fore Biotherapeutics is a clinical-stage biotechnology company focused on developing precision oncology treatments tailored to patients with specific genetic mutations. By utilizing functional genomics, the company aims to create a pipeline of therapies that address unmet needs in cancer care. Fore Biotherapeutics conducts research to replicate naturally occurring mutations in vitro, allowing for the assessment of their impact on cellular signaling pathways and responses to various compounds. This approach not only facilitates the identification of novel target mutations but also helps biopharma companies broaden the patient subgroups that may benefit from existing cancer therapies.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

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

Vicore Pharma is a clinical-stage pharmaceutical company dedicated to developing innovative treatments for severe lung disorders, particularly idiopathic pulmonary fibrosis (IPF). The company's lead drug candidate, C21, is a small molecule angiotensin II type 2 receptor agonist currently in phase 2a development and has received orphan drug designation in both the EU and the US. C21 is also being explored for potential applications in other rare diseases associated with the AT2 receptor. In addition to its pharmacological efforts, Vicore Pharma is developing Almee, an investigational medical device that employs cognitive behavioral therapy to address the psychological challenges faced by patients with pulmonary fibrosis. The company operates from Astra Zeneca’s Bioventurehub in Mölndal.

Upstream Bio is a clinical-stage biotechnology company dedicated to developing antibody therapies for inflammatory diseases, particularly severe asthma and other respiratory disorders. The company's lead candidate, verekitug, is a monoclonal antibody that acts as an antagonist to the Thymic Stromal Lymphopoietin (TSLP) receptor. TSLP is a key cytokine that drives inflammatory responses and is strategically positioned upstream of various signaling pathways influencing immune cells. Upstream Bio aims to address unmet medical needs in the treatment of severe respiratory conditions by leveraging this validated target to create effective therapies.

Convergent Therapeutics is a clinical-stage biotechnology company focused on developing innovative radiopharmaceutical therapies specifically for prostate cancer. The company utilizes a proprietary dual-targeted radionuclide therapy technology, initially developed by Dr. Neil Bander from Weill Cornell Medicine, which is licensed from Cornell University. Its lead product, CONV 01-α, is a monoclonal antibody conjugated with the radioactive alpha particle emitter 225Ac. This therapy is designed to bind to the prostate-specific membrane antigen (PSMA) and, upon binding, internalize to deliver a potent radioactive payload directly into prostate cancer cells. CONV 01-α is protected by multiple U.S. and foreign patents, and if approved by the FDA, it would be the first antibody to deliver a radioisotope specifically for prostate cancer treatment, marking a significant advancement in cancer therapy. Convergent Therapeutics aims to explore dual-targeted combination strategies to enhance cancer treatment efficacy, leveraging various targeting agents and therapeutic doses.

Aer Therapeutics is a clinical-stage biopharmaceutical company that develops an approach to treat lung disease with excess mucus and mucus plugs. The company focuses on treating respiratory diseases with significant unmet needs, such as COPD, cystic fibrosis, and asthma.

Harpoon Therapeutics is a clinical-stage immunotherapy company developing a novel class of T cell engagers that harness the power of the body’s immune system to treat patients suffering from cancer and other diseases. T cell engagers are engineered proteins that direct a patient’s own T cells to kill target cells that express specific proteins, or antigens, carried by the target cells. Using its proprietary Tri-specific T cell Activating Construct (TriTAC), platform, Harpoon is developing a pipeline of novel T cell engagers, or TriTACs, initially focused on the treatment of solid tumors and hematologic malignancies. Harpoon's first drug candidate, HPN424, is currently in Phase 1 clinical trial as a treatment for prostate cancer.

Congruence Therapeutics operates as a biotechnology company working at the interface of computational and experimental drug discovery to design novel small molecules for diseases of protein misfolding.

BiomX discovers and develops innovative microbiome therapeutics. The Company's mission is to develop novel therapeutics for preventing and treating diseases in which microbiome imbalances have been implicated. BiomX's microbiome modulation technologies are based on the cutting-edge innovative research of its scientific collaborators: Professor Rotem Sorek, PhD and Dr. Eran Elinav, MD, PhD both of the Weizmann Institute of Science and Professor Timothy K. Lu, MD, PhD of the Massachusetts Institute of Technology.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Pathalys is a clinical biopharmaceutical company that develops therapeutics that improve late-stage chronic kidney disease. Pathalys' initial focus will be on improving treatment for secondary hyperparathyroidism, which is an unmet need (SHPT). SHPT is a condition in which a disease outside of the parathyroid glands causes all of the parathyroid glands to swell and become hyperactive, producing an excessive amount of parathyroid hormone (PTH).

Amolyt Pharma is a clinical-stage biotechnology company focused on developing peptide-based therapies for rare endocrine and metabolic disorders. The company's key programs include AZP-3601, a parathyroid hormone analog aimed at treating hypoparathyroidism by maintaining sustained calcium levels in the blood, and AZP-3404, a novel peptide designed to improve fat and glucose metabolism. By addressing severe insulin resistance and other critical metabolic conditions, Amolyt Pharma aims to provide effective treatment options for patients facing these rare disorders.

Stairway Medical is a ladder of human progress and evolution stairs up for human health. The technology and products will be applicable to medical scenarios such as human function restoration, disease treatment, and functional enhancement, as well as related scientific research work.

MBX Biosciences, Inc. is a biotechnology company based in Carmel, Indiana, specializing in the development of therapeutics for rare endocrine disorders. Established in 2018, the company focuses on creating peptides aimed at treating genetic diseases that affect glandular hormones, which play a crucial role in the body’s chemical messaging system. By targeting conditions with inadequate treatment options, MBX Biosciences is dedicated to discovering, developing, and commercializing innovative endocrine therapeutics.

Pulnovo Medical, founded in 2013 in Wuxi, Anhui, specializes in the development, production, and service of innovative medical devices aimed at treating pulmonary hypertension. The company focuses on advanced pulse radiofrequency ablation technology, which includes pioneering high-frequency ablation equipment and intravascular catheters. These products are designed to enhance the quality of life for patients suffering from pulmonary hypertension and heart failure, offering a novel approach to treatment with a commitment to improving patient outcomes. Pulnovo Medical holds global invention patents for its unique technologies, underscoring its role as a leader in this specialized medical field.

Alpheus Medical specializes in developing innovative treatment solutions for solid body cancers, focusing primarily on aggressive brain tumors. The company has created a novel sonodynamic therapy platform that employs a non-invasive drug-device combination for the outpatient treatment of recurrent glioblastoma multiforme (rGBM). This approach utilizes photodynamic therapy, which involves using light delivered through a minimally invasive device to target cancer cells that have been exposed to a photosensitizer. By offering these advanced therapeutic options, Alpheus Medical aims to provide effective alternatives to traditional invasive brain surgeries, enhancing patient care and treatment outcomes.

Compass Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing therapeutic antibodies for solid tumors and hematological malignancies. Founded in 2014 and based in Cambridge, Massachusetts, the company utilizes its proprietary StitchMabs and common light-chain platforms to identify and develop innovative antibody therapeutics. Its lead product candidate, CTX-471, is an agonistic monoclonal antibody targeting CD137, currently undergoing Phase I clinical trials. Another significant candidate, CTX-009, is a bispecific antibody that inhibits DLL4-mediated Notch signaling and VEGF-A signaling, and has completed a Phase 1 dose escalation study. Additionally, CTX-8371, another bispecific antibody targeting PD-1 and PD-L1, is in the IND-enabling stage. Compass Therapeutics aims to engage the immune system effectively to advance its therapeutic offerings.

Alterome Therapeutics is a biotechnology company focused on precision oncology, dedicated to developing targeted therapeutics that specifically address validated oncogenic drivers in cancer. The firm aims to discover and create alteration-specific therapies that provide hope and improved treatment options for individuals affected by cancer. By concentrating on high-value oncogenic alterations, Alterome Therapeutics seeks to enhance the effectiveness of cancer treatments and support healthcare providers in their efforts to combat the disease.

Insmed Incorporated is a global biopharmaceutical company dedicated to improving the lives of patients with serious and rare diseases. Its flagship product, ARIKAYCE (amikacin liposome inhalation suspension), is approved in the United States for treating Mycobacterium avium complex (MAC) lung disease, targeting adult patients who have limited or no alternative treatment options. MAC lung disease is a rare and potentially fatal infection that can lead to significant lung damage. In addition to ARIKAYCE, Insmed's clinical pipeline features Brensocatib, a novel oral reversible inhibitor of dipeptidyl peptidase 1, which shows promise for treating non-cystic fibrosis bronchiectasis and other inflammatory conditions. Another candidate in development is INS1009, an inhaled formulation of a treprostinil prodrug aimed at addressing rare pulmonary disorders, including pulmonary arterial hypertension.

PIC Therapeutics, Inc. is a biotechnology company dedicated to innovating cancer treatment through the selective modulation of oncogene translation. Founded in 2016 and based in Boston, Massachusetts, the company focuses on developing therapeutics that target the "master switch" of cancer signaling pathways. By modulating the pre-initiation complex responsible for messenger RNA translation, PIC Therapeutics aims to selectively block the production of oncogene proteins. This precision-based approach has the potential to simultaneously address multiple oncogenic drivers, thereby improving patient outcomes and advancing a new generation of cancer treatments.

Treeline Biosciences is a biotechnology company established in 2021 and based in Stamford, Connecticut. The company focuses on developing transformative precision medicines aimed at treating cancer and other serious health conditions. Utilizing a comprehensive drug discovery platform, Treeline combines mechanistic biology, medicinal chemistry, computational science, structural biology, and protein science to create innovative therapeutic solutions. Through its research and development efforts, Treeline aims to provide effective treatments to improve patient outcomes in oncology and beyond.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

ACELYRIN is a biopharma company focused on providing patients life-changing new treatment options by identifying, acquiring, and accelerating the development and commercialization of promising drug candidates and by leveraging its expertise to rapidly advance these medicines to patients.

Adaptive Biotechnologies is a commercial-stage company focused on advancing immune-driven medicine by leveraging the adaptive immune system's biology to enhance disease diagnosis and treatment. Founded in 2009 and headquartered in Seattle, Washington, the company offers the clonoSEQ diagnostic test, which is authorized by the FDA for detecting and monitoring minimal residual disease in patients with specific blood cancers, including multiple myeloma and certain forms of leukemia. Additionally, Adaptive provides immunoSEQ services and kits for research purposes, aiding in the discovery of prognostic and diagnostic signals. The company is engaged in strategic collaborations with organizations such as Genentech and Microsoft to develop innovative therapies and diagnostic tools. Its offerings cater to life sciences research, clinical diagnostics, and drug discovery sectors.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

MiroBio's platform is based on ground-breaking research by their scientific founders at the University of Oxford delineating how immune cells communicate and how they are activated. These insights have enabled MiroBio to create antibodies that can leverage natural signalling mechanisms to restore immune system balance and control.

Frontera Therapeutics is a company specializing in adeno-associated virus (AAV) gene therapy, focused on developing and commercializing cost-effective recombinant virus-based therapies. The company aims to create a scalable platform that produces high-quality, affordable rAAV therapies for a range of genetic disorders, including ophthalmic, liver, metabolic, and neuromuscular diseases. Frontera Therapeutics has a diverse product pipeline that encompasses multiple therapeutic areas, with various candidates at different stages of development. By prioritizing affordability and quality, the company seeks to enhance the lives of patients through innovative genetic therapies.

Aerin Medical Inc. is a health technology company based in Sunnyvale, California, specializing in non-invasive treatment solutions for nasal breathing disorders. Established in 2011, the company has developed therapeutic devices that utilize low-power radiofrequency energy to remodel nasal soft tissues, effectively addressing the underlying causes of nasal congestion. Its primary products include the VivAer procedure for nasal airway obstruction and the RhinAer procedure for chronic rhinitis, both of which employ proprietary temperature-controlled technology. These solutions offer ENT physicians appealing alternatives to invasive surgery, allowing them to treat patients under local anesthesia with minimal to no downtime. Aerin Medical aims to meet the unmet needs of individuals suffering from chronic nasal airway conditions through its innovative and office-friendly product offerings.

CHARM Therapeutics is focused on developing innovative medicines using advanced 3D deep learning and drug discovery technologies. The company's platform aims to address undruggable disease targets, providing healthcare professionals with effective treatment options for challenging medical conditions. By leveraging cutting-edge technology, CHARM Therapeutics seeks to create transformative therapies that have the potential to significantly improve patient outcomes.

Upstream Bio is a clinical-stage biotechnology company dedicated to developing antibody therapies for inflammatory diseases, particularly severe asthma and other respiratory disorders. The company's lead candidate, verekitug, is a monoclonal antibody that acts as an antagonist to the Thymic Stromal Lymphopoietin (TSLP) receptor. TSLP is a key cytokine that drives inflammatory responses and is strategically positioned upstream of various signaling pathways influencing immune cells. Upstream Bio aims to address unmet medical needs in the treatment of severe respiratory conditions by leveraging this validated target to create effective therapies.

Terremoto Biosciences is a biotechnology company leveraging the power of lysine-based covalency to develop optimized medicines.

Odyssey Therapeutics is a biotechnology company focused on developing next-generation immunomodulators and oncology drugs. By leveraging an integrated drug-hunting engine, the company aims to enhance the efficiency of drug discovery, identifying promising targets while reducing the likelihood of failure during development. Odyssey Therapeutics is committed to advancing the field of drug discovery and expediting clinical development, ultimately contributing to the creation of more effective precision medicines for the healthcare industry.

Aspen Neuroscience, Inc. is a biotechnology company focused on developing induced pluripotent stem cells (iPSCs) to create patient-specific therapies for neurological conditions, particularly Parkinson's disease. Founded in 2018 and based in San Diego, California, the company is advancing its pipeline of products, which includes ANPD001, an autologous neuron replacement therapy for sporadic Parkinson's disease, and ANPD002, a gene-edited autologous neuron replacement therapy targeting familial forms of the disease. Aspen Neuroscience employs innovative genomic approaches in conjunction with stem cell biology to address diseases with significant unmet medical needs, aiming to provide restorative cell therapies that can modify the progression of Parkinson's disease and potentially extend to other affected organs.

Valneva is a pharmaceutical company specializing in the development of prophylactic vaccines aimed at addressing infectious diseases with significant unmet medical needs. The company has successfully commercialized several vaccines, including IXIARO/JESPECT, DUKORAL, and IXCHIQ, primarily targeting travelers. In addition to its marketed products, Valneva is advancing a diverse pipeline of vaccine candidates, including those for Lyme disease, chikungunya, and COVID-19. The company is involved in the entire lifecycle of vaccine development, encompassing research, manufacturing, and distribution. Valneva also offers technologies and services aimed at enhancing vaccine commercialization, which further supports its mission to deliver life-saving immunizations globally.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

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

ArriVent Biopharma is a clinical-stage biopharmaceutical company focused on developing innovative treatments for currently untreatable cancers. The company is committed to addressing unmet medical needs in oncology by leveraging its team's extensive drug development experience. ArriVent aims to maximize the potential of its lead candidate, furmonertinib, while advancing a pipeline of novel therapeutics, including next-generation antibody-drug conjugates. With an initial emphasis on solid tumors, ArriVent seeks to bring differentiated medicines from development through to commercialization, ultimately improving patient outcomes in cancer treatment.

Congruence Therapeutics operates as a biotechnology company working at the interface of computational and experimental drug discovery to design novel small molecules for diseases of protein misfolding.

HanBio Therapeutics is a biotechnology company committed to researching and developing next generation transformative medicines.

ImmPACT Bio USA Inc. is a cell therapy company focused on developing engineered T-cells specifically designed to target and treat solid tumors. By utilizing advanced technology, the company aims to create chimeric antigen receptor (CAR) T-cell therapies that exhibit tumor specificity, allowing them to induce cytotoxicity only when interacting with tumor cells while sparing healthy tissues. This selective targeting mitigates the safety risks commonly associated with existing CAR-T therapies, providing a promising approach to cancer treatment that enhances efficacy and reduces potential harm to noncancerous cells.

Alterome Therapeutics is a biotechnology company focused on precision oncology, dedicated to developing targeted therapeutics that specifically address validated oncogenic drivers in cancer. The firm aims to discover and create alteration-specific therapies that provide hope and improved treatment options for individuals affected by cancer. By concentrating on high-value oncogenic alterations, Alterome Therapeutics seeks to enhance the effectiveness of cancer treatments and support healthcare providers in their efforts to combat the disease.

Nucleix Ltd. is a company that develops, manufactures, and markets non-invasive molecular cancer diagnostic tests. Founded in 2008 and headquartered in Rehovot, Israel, with an additional office in San Diego, California, Nucleix focuses on identifying subtle changes in DNA methylation patterns to enhance cancer detection. The company's key products include Bladder EpiCheck, a urine test designed for monitoring bladder cancer, and Lung EpiCheck, a blood test aimed at the early detection of lung cancer. By leveraging proprietary biomarker assays, Nucleix enables healthcare providers to improve treatment outcomes through more accurate cancer screening and monitoring. The company has established distribution partnerships in several countries, including Israel, the Netherlands, Turkey, Russia, Italy, and Denmark.

Leal Therapeutics is a Massachusetts-based company focused on developing innovative therapeutics for patients with significant central nervous system conditions. The company addresses urgent and unmet medical needs by providing precision medicines tailored to improve patient outcomes. Through its dedication to advancing therapeutic services, Leal Therapeutics aims to enhance the quality of care for individuals affected by challenging neurological disorders.

Emergence Therapeutics AG is a biopharmaceutical company based in Duisburg, Germany, focused on developing novel antibody-drug conjugate (ADC) immuno-therapeutics for high-need cancers. Founded in 2019, the company is advancing its lead program, which combines a highly specific antibody with optimized linker and payload technology to target Nectin-4, a clinically validated target for various cancers. This approach is inspired by the success of enfortumab vedotin, an approved treatment for urothelial cancers. Emergence Therapeutics is also exploring opportunities to create additional first- or best-in-class ADCs, aiming to address significant therapeutic needs in oncology.

Cyrus Biotechnology, Inc. specializes in developing software tools for protein structure prediction and design, aiming to enhance research in biotechnology and pharmaceuticals. The company's flagship product, Rosetta, allows for the design of biologically active proteins that can address critical health issues, such as brain cancer treatment and gluten breakdown in Celiac Disease patients. Additionally, Cyrus Bench offers an enterprise version of the Rosetta toolkit, equipped with a variety of bio-molecular computation tools. The company also provides Cyrus CryoEM services, which include advanced structure refinement and model building. Founded in 2014 and headquartered in Seattle, Washington, Cyrus Biotechnology serves a diverse clientele, including pharmaceutical companies, venture-backed startups, and mid-sized biotech firms focused on therapeutic discovery and industrial biotechnology innovation.

Alpheus Medical specializes in developing innovative treatment solutions for solid body cancers, focusing primarily on aggressive brain tumors. The company has created a novel sonodynamic therapy platform that employs a non-invasive drug-device combination for the outpatient treatment of recurrent glioblastoma multiforme (rGBM). This approach utilizes photodynamic therapy, which involves using light delivered through a minimally invasive device to target cancer cells that have been exposed to a photosensitizer. By offering these advanced therapeutic options, Alpheus Medical aims to provide effective alternatives to traditional invasive brain surgeries, enhancing patient care and treatment outcomes.

ACELYRIN is a biopharma company focused on providing patients life-changing new treatment options by identifying, acquiring, and accelerating the development and commercialization of promising drug candidates and by leveraging its expertise to rapidly advance these medicines to patients.

Capstan Therapeutics is a biotechnology company focused on enhancing the therapeutic potential of in vivo RNA-based therapies. It develops proprietary targeted delivery systems, including targeted lipid nanoparticles, to improve the efficacy and control of dosage in engineered cell therapies. This innovative platform aims to address a variety of diseases, particularly in the fields of oncology, fibrosis, and inflammation, where there are significant unmet clinical needs. By advancing precision in vivo cell engineering, Capstan Therapeutics seeks to create transformative therapeutics that can offer new solutions for patients facing these challenging conditions.

ReCode Therapeutics is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company employs a unique non-viral lipid nanoparticle platform that facilitates the precise delivery of nucleic acid and protein payloads to specific organs and tissues, enhancing the effectiveness of genetic therapies. This selective organ targeting (SORT) technology allows for the administration of corrective or disease-modifying treatments beyond the liver, addressing the challenges faced by traditional gene therapy methods. By harnessing these innovative techniques, ReCode Therapeutics aims to provide new therapeutic options for patients suffering from rare and life-limiting respiratory diseases.

Garuda Therapeutics is a biotech company focused on developing off-the-shelf hematopoietic stem cell therapies aimed at treating life-threatening diseases, particularly those affecting blood, bone marrow, immunity, and metabolism. The company is dedicated to eliminating the reliance on donor or patient cells for blood stem cell transplants by leveraging its innovative platform technology to generate self-renewing blood stem cells. This approach is designed to ensure rapid and broad access to consistent, durable, HLA-compatible, and transgene-free blood stem cell therapies for patients. Garuda Therapeutics employs advanced methodologies, including the use of zebrafish, mice, and human-induced pluripotent stem cells, to clone and analyze novel genes and mechanisms critical for blood formation, ultimately developing mechanism-based therapies that address unmet medical needs.

Ventyx Biosciences, Inc. is a clinical-stage biopharmaceutical company based in Encinitas, California, that specializes in developing selective inhibitors of TYK2 for autoimmune diseases. Founded in 2018, the company focuses on creating innovative therapies for patients suffering from inflammatory diseases and autoimmune disorders. Its pipeline includes VTX958, an oral allosteric TYK2 inhibitor currently in Phase 1 trials, aimed at treating a variety of autoimmune conditions while minimizing the toxicities associated with broader Janus kinase inhibition. Additionally, Ventyx is advancing VTX002, a Phase 2-ready S1P1 receptor modulator for ulcerative colitis, and VTX2735, a Phase 1 peripheral inhibitor of the NLRP3 inflammasome, which is implicated in several inflammatory conditions. Through these efforts, Ventyx aims to provide effective treatment options for millions of patients.

Amolyt Pharma is a clinical-stage biotechnology company focused on developing peptide-based therapies for rare endocrine and metabolic disorders. The company's key programs include AZP-3601, a parathyroid hormone analog aimed at treating hypoparathyroidism by maintaining sustained calcium levels in the blood, and AZP-3404, a novel peptide designed to improve fat and glucose metabolism. By addressing severe insulin resistance and other critical metabolic conditions, Amolyt Pharma aims to provide effective treatment options for patients facing these rare disorders.

Vanqua Bio is a biotechnology company dedicated to discovering and developing innovative medicines for patients suffering from neurodegenerative diseases. The company leverages a technology platform that incorporates human genetics and patient-derived neuronal cells to identify and validate new disease pathways linked to lysosomal dysfunction and the inappropriate activation of the innate immune system. Vanqua Bio focuses on creating small-molecule activators of glucocerebrosidase and advancing programs that target the innate immune system, with the aim of addressing the progression of various neurological disorders. Through its efforts, the company seeks to provide healthcare professionals with transformative treatment options for patients facing these challenging conditions.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.