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Latigo Biotherapeutics is a clinical-stage biotechnology company that develops novel, non-opioid therapies for chronic pain. The company aims to target pain at its source to provide effective, rapid-acting pain relief without the risk of addiction. Their lead program targets Nav1.8, a validated human pain target, leveraging internal ion channel expertise.

Aclaris Therapeutics is a United States-based clinical-stage biopharmaceutical company focused on developing novel therapies for immuno-inflammatory diseases and dermatology, aiming to address unmet medical and aesthetic needs where treatment options are limited. The company operates two segments, therapeutics and contract research, with the therapeutics business pursuing innovative treatments for immuno-inflammatory conditions while the contract research segment provides laboratory services. Its pipeline includes Zunsemetinib, an oral MK2 inhibitor, and ATI-2138, among other drug candidates.

City Therapeutics is a biopharmaceutical company specializing in the development of next-generation small-interfering RNAs (siRNAs) to enhance RNA interference (RNAi) therapies. The company's pipeline comprises innovative RNAi therapeutics targeting diverse disease indications. City Therapeutics aims to improve and expand RNAi technology to achieve better patient outcomes, led by a team of experienced scientists and industry professionals committed to advancing the field of RNA interference.

ArsenalBio is a programmable cell therapy company established in 2019, focused on developing and commercializing innovative immune cell therapies, particularly for cancer treatment. The company utilizes advanced technologies, including CRISPR-based genome engineering, synthetic biology, and machine learning, to create multifunctional T-cell therapies. ArsenalBio's approach involves the precise insertion of large synthetic DNA sequences, enabling the generation of next-generation autologous T-cell therapies. By integrating these cutting-edge methods, ArsenalBio aims to enhance the effectiveness and safety of immune cell therapies, reduce costs for healthcare providers, and improve market access, ultimately broadening patient outcomes and making these therapies more widely available.

Founded in 2015, Imperative Care specializes in developing innovative medical technologies aimed at improving stroke treatment. Its flagship products include the Large Distal Platform catheter for deeper reach and the ZOOM Aspiration System for clot removal. The company's mission is to enhance treatment speed and effectiveness, ultimately optimizing patient recovery.

Mereo Biopharma, established in London in 2015, specializes in acquiring and advancing late-stage drug candidates from larger pharmaceutical companies. These candidates, often overlooked due to resource constraints, target rare and specialized diseases with significant unmet medical needs. Mereo's strategy involves rapidly progressing these assets through clinical development, either by partnering or divesting them, or directly commercializing them, particularly in orphan disease indications. The company combines operational efficiency with financial resources, leveraging a unique partnership with a leading global CRO, ICON, to conduct comprehensive clinical studies. Mereo's focus is on transforming the lives of patients worldwide by developing innovative medicines that can significantly improve their quality of life.

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

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

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.

Leap Therapeutics is a clinical-stage biopharmaceutical company focused on developing targeted and immuno-oncology therapeutics. Its primary focus is to advance translational-stage molecules in cell signaling and immuno-oncology areas.

Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies, utilizing its proprietary technology to maximize the effectiveness of immuno-oncology treatments while minimizing side effects outside the tumor. Among its key products is XTX201, which aims to induce immune activity directly within tumors, demonstrating improved antitumor effects. Founded in 2015 and based in Waltham, Massachusetts, Xilio Therapeutics was previously known as Akrevia Therapeutics until its rebranding in March 2020. The company is dedicated to advancing a pipeline of novel immuno-oncology molecules, including tumor-activated cytokines and antibodies, with the goal of significantly improving patient outcomes in cancer treatment.

Founded in 2019, Enliven Therapeutics is a precision oncology company based in Boulder, Colorado. It focuses on developing small molecule therapies to extend and improve patient lives by targeting clinically validated biological targets with industry-leading chemistry.

FogPharma is a biotechnology company focused on developing innovative therapies for cancer treatment through its proprietary cell-penetrating mini proteins (CPMPs). Founded by Greg Verdine, a leader in drug discovery, the company aims to target cancer-causing proteins within cells that are typically inaccessible to traditional drugs. This novel approach allows for the neutralization of these proteins, potentially transforming the treatment landscape for cancer-related diseases. FogPharma collaborates with leading experts in cancer biology and therapy, fostering a network of researchers, investors, and cancer patients dedicated to advancing its mission. The company is committed to delivering this new class of medicines to improve patient outcomes and enhance quality of life for individuals affected by cancer.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

Jasper Therapeutics, Inc. is a clinical-stage biotechnology company based in Menlo Park, California, that specializes in developing therapeutic agents for hematopoietic stem cell transplants and gene therapies. Founded in 2018, the company aims to enhance the safety and efficacy of conditioning agents to broaden the application of curative therapies. Jasper's lead product, JSP191, is a humanized monoclonal antibody currently in clinical development, designed to act as a conditioning agent by selectively clearing hematopoietic stem cells from bone marrow in patients undergoing stem cell transplants. The company also utilizes an engineered hematopoietic stem cells platform to address the limitations of transplant grafts, thereby increasing the accessibility of both allogeneic and autologous gene-edited hematopoietic stem cell therapies for a larger patient population.

Apollo Therapeutics is a collaborative biopharmaceutical venture bridging three UK universities—Imperial College London, University College London, and the University of Cambridge—and three global pharmaceutical companies—AstraZeneca, GlaxoSmithKline, and Johnson & Johnson Innovation. It provides translational funding and drug discovery expertise to translate fundamental academic research into medicines, supporting preclinical and clinical development. The company builds an expansive, de-risked portfolio across oncology, major inflammatory disorders, and rare diseases, focusing on assets with strong biological hypotheses. Its team of industry scientists works with academics and industry partners to tailor projects, aiming to accelerate the path from discovery to the clinic and improve patient outcomes. By leveraging the UK science base and domestic pharma capabilities, Apollo Therapeutics seeks to deliver new therapies that address significant medical needs.

Avalyn Pharma is a biopharmaceutical company focused on developing therapies for severe respiratory diseases, notably idiopathic pulmonary fibrosis (IPF). Established in 2011, the company is headquartered in Seattle, Washington.

Apollo Therapeutics is a collaborative biopharmaceutical venture bridging three UK universities—Imperial College London, University College London, and the University of Cambridge—and three global pharmaceutical companies—AstraZeneca, GlaxoSmithKline, and Johnson & Johnson Innovation. It provides translational funding and drug discovery expertise to translate fundamental academic research into medicines, supporting preclinical and clinical development. The company builds an expansive, de-risked portfolio across oncology, major inflammatory disorders, and rare diseases, focusing on assets with strong biological hypotheses. Its team of industry scientists works with academics and industry partners to tailor projects, aiming to accelerate the path from discovery to the clinic and improve patient outcomes. By leveraging the UK science base and domestic pharma capabilities, Apollo Therapeutics seeks to deliver new therapies that address significant medical needs.

Founded in 2019, Genesis Therapeutics is a biotechnology company based in South San Francisco, California. It specializes in developing small molecule drug candidates for treating debilitating disorders using artificial intelligence-driven platforms.

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

Hemab is a company that creates preventative treatments for bleeding and thrombosis problems. The medicines developed by the business aggregate, modify, balance, and at times recruit endogenous clotting components already present in the blood to the site of injury, allowing patients to produce hemostatic plugs or clots to avert life-threatening complications. Hemab Therapeutics was established in 2017 in Copenhagen, Hovedstaden by Johan H. Faber and Mads Behrndt.

Spruce Biosciences is a biopharmaceutical company focused on developing therapies for rare endocrine disorders. Its pipeline includes tildacerfont for classic congenital adrenal hyperplasia in adults and children, with ongoing clinical development, and Tralesinidase Alfa in its portfolio. The company collaborates with Eli Lilly to research, develop, and commercialize compounds. Founded in 2014, Spruce Biosciences is headquartered in Daly City, California, and aims to deliver meaningful therapies for patients with rare endocrine diseases.

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

Treeline Biosciences is a biotechnology company focused on developing precision medicines for cancer and other serious diseases. Founded in 2021 and based in Stamford, Connecticut, the company uses a platform that integrates pathophysiology, genomics, and pattern recognition to identify therapeutic approaches that address unmet patient needs.

Kymera Therapeutics is a biotechnology company focused on targeted protein degradation. It employs a proprietary predictive modeling platform to discover novel small molecule therapeutics that selectively degrade disease-causing proteins.

Immunocore is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

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

Mineralys Therapeutics, Inc. is a clinical-stage biopharmaceutical company focused on developing innovative therapies for hypertension. The company is advancing its lead product candidate, MLS-101, a highly selective and potent aldosterone synthase inhibitor licensed from Mitsubishi Tanabe Pharma Corporation. Mineralys aims to provide a targeted treatment for patients with uncontrolled hypertension, particularly those who do not achieve adequate blood pressure control despite using multiple antihypertensive medications. By addressing the underlying factors associated with elevated aldosterone levels, Mineralys seeks to improve treatment outcomes for individuals suffering from this condition.

Founded in 2019 and based in Cambridge, Massachusetts, MoMa Therapeutics discovers precision medicines by targeting molecular machines underlying human diseases. The company's platform enables drug development from previously intractable enzyme classes.

MiRXES Pte. Ltd., founded in 2014 and based in Singapore, specializes in life science research tools and molecular diagnostic tests for both research and clinical applications. The company has developed a leading microRNA (miRNA) detection assay platform designed to identify cancer at asymptomatic stages. Its product offerings include RT-qPCR assays, assay panels, spike-in RNA kits, and solutions for biomarker discovery. By focusing on early cancer detection, MiRXES provides healthcare professionals with essential diagnostic tools to improve clinical outcomes through timely intervention.

Simcha Therapeutics is a biopharmaceutical company focused on developing engineered cytokine immunotherapy for cancer treatment. Founded in 2018 and headquartered in New Haven, Connecticut, the company utilizes directed evolution to create novel cytokines that enhance the immune system's ability to combat diseases. Its lead program, ST-067, is a designer IL-18 cytokine that has demonstrated significant antitumor effects in animal models, both as a standalone treatment and in combination with anti-PD-1 checkpoint inhibitors. This innovative approach aims to provide patients with more effective immunotherapies, thereby improving their ability to fight cancer.

AvengeBio is a biotechnology company based in Cambridge, Massachusetts, focused on developing cell-generated immunotherapies aimed at eradicating solid tumors. Founded in 2019, the company is advancing a drug delivery platform designed to enable the precision delivery of cytokines and other potent immunomodulatory molecules. This innovative approach allows for controlled release over time, fostering robust local and systemic anti-tumor immune responses while minimizing the toxicities typically associated with conventional immunotherapies. Through its research and development efforts, AvengeBio seeks to address the challenges posed by intractable solid tumors and improve treatment outcomes for cancer patients.

EQRx, Inc. is a biotechnology company based in Cambridge, Massachusetts, established in 2019. The company focuses on re-engineering the drug discovery and delivery process to provide a market-based solution to the escalating costs of medications. EQRx aims to enhance the efficiency and affordability of high-quality, patent-protected medicines by collaborating with various stakeholders across the healthcare system and leveraging advancements in science and technology. The company has developed a robust pipeline with over ten programs, including clinical and preclinical projects targeting oncology and immune-inflammatory conditions, featuring compounds such as Aumolertinib, Lerociclib, Sugemalimab, and Nofazinlimab.

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, founded in 2018. The company specializes in the development of innovative treatments for cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at addressing resistant hypertension and primary aldosteronism. By advancing its clinical candidates toward marketing approval, CinCor seeks to provide effective solutions for patients suffering from these conditions.

Aadi Bioscience is a clinical-stage biopharmaceutical company developing precision therapies for cancers driven by mTOR pathway alterations. It focuses on targeted mTOR inhibitors to treat genetically defined cancers, including alterations in TSC1 or TSC2 genes, where conventional inhibitors have struggled with pharmacology, delivery, safety, or tumor targeting. The company is advancing ABI-009, a clinical-phase mTOR inhibitor intended for oncology indications as well as cardiovascular and metabolic contexts. Aadi aims to bring transformative therapies to patients with ultra-rare cancers such as PEComa, and it traces its origins to an earlier name, with its operations based in Pacific Palisades, California, and a founding year of 2011.

Vigil Neuroscience is a clinical-stage biotechnology company focused on developing therapeutics that restore the function of microglia, the brain's immune cells. The company aims to improve outcomes for patients with neurodegenerative diseases by advancing its lead monoclonal antibody TREM2 agonist through Phase 1 trials and progressing its small molecule TREM2 agonist towards an Investigational New Drug (IND) application.

Vedanta Biosciences is a clinical-stage microbiome company focused on developing therapies for immune-mediated and infectious diseases using live bacteria derived from the human microbiome. The company designs orally delivered defined bacterial consortia, leveraging libraries of microbiome-derived strains, proprietary datasets from human interventional studies, and tools for selecting potent strains. Its platform encompasses consortium design and CGMP manufacturing of bacterially defined products, enabling physicians to access live bacteria drugs for autoimmune and inflammatory conditions. Headquartered in Cambridge, Massachusetts, Vedanta Biosciences aims to create a new category of oral therapies arising from defined bacterial communities grown from clonal cell banks.

Amylyx Pharmaceuticals, Inc. is a biotechnology company focused on developing innovative therapies for neurodegenerative diseases, particularly Amyotrophic Lateral Sclerosis (ALS), Alzheimer’s disease, and Wolfram syndrome. Founded in 2013 and based in Cambridge, Massachusetts, the company is best known for its investigational therapy AMX0035, a fixed-dose co-formulation of sodium phenylbutyrate and Taurursodiol. This treatment aims to address the energy crisis within mitochondria and the accumulation of toxic, unfolded proteins in the endoplasmic reticulum, both of which contribute to the progression of neurodegenerative conditions. Through its research and development efforts, Amylyx seeks to provide effective solutions to mitigate the suffering caused by these debilitating diseases.

Founded in 2015, Imperative Care specializes in developing innovative medical technologies aimed at improving stroke treatment. Its flagship products include the Large Distal Platform catheter for deeper reach and the ZOOM Aspiration System for clot removal. The company's mission is to enhance treatment speed and effectiveness, ultimately optimizing patient recovery.

MiRXES Pte. Ltd., founded in 2014 and based in Singapore, specializes in life science research tools and molecular diagnostic tests for both research and clinical applications. The company has developed a leading microRNA (miRNA) detection assay platform designed to identify cancer at asymptomatic stages. Its product offerings include RT-qPCR assays, assay panels, spike-in RNA kits, and solutions for biomarker discovery. By focusing on early cancer detection, MiRXES provides healthcare professionals with essential diagnostic tools to improve clinical outcomes through timely intervention.

Apollo Therapeutics is a collaborative biopharmaceutical venture bridging three UK universities—Imperial College London, University College London, and the University of Cambridge—and three global pharmaceutical companies—AstraZeneca, GlaxoSmithKline, and Johnson & Johnson Innovation. It provides translational funding and drug discovery expertise to translate fundamental academic research into medicines, supporting preclinical and clinical development. The company builds an expansive, de-risked portfolio across oncology, major inflammatory disorders, and rare diseases, focusing on assets with strong biological hypotheses. Its team of industry scientists works with academics and industry partners to tailor projects, aiming to accelerate the path from discovery to the clinic and improve patient outcomes. By leveraging the UK science base and domestic pharma capabilities, Apollo Therapeutics seeks to deliver new therapies that address significant medical needs.

Synthekine Inc. is a biotechnology company based in Menlo Park, California, specializing in the development of engineered cytokine therapies and immunotherapies aimed at treating cancer and autoimmune disorders. Founded in 2018, Synthekine utilizes advanced platform technologies, including engineered partial agonists and orthogonal cell therapies, to enhance the efficacy of cytokines while minimizing their toxic side effects. The company's innovative Synthekine Platform employs surrogate agonists instead of traditional mutant cytokines to create selective immunotherapies. Its product pipeline features candidates such as STK-009, an orthogonal ligand paired with SYNCAR-001, an orthogonal receptor-modified CAR-T therapy targeting CD-19. Additionally, Synthekine is developing STK-012, partial agonists of IL-2, and other novel immunotherapies that integrate cytokine receptors to initiate new signaling pathways without relying on wild-type cytokines.

Caris Life Sciences specializes in advanced molecular profiling technologies for cancer diagnostics and drug development. Established in 1987, it offers prognostic testing services, genomic profiling, and clinico-genomic data platforms to improve patient outcomes. The company operates globally with offices in the US and Switzerland.

Theseus Pharmaceuticals is a biopharmaceutical company dedicated to enhancing the lives of cancer patients through the development of targeted therapies. The company focuses on discovering and commercializing innovative treatments that specifically address drug resistance mutations in critical driver oncogenes, which are genes that, when mutated, contribute to cancer progression. By leveraging advanced technology, Theseus Pharmaceuticals aims to transform cancer care and improve patient outcomes.

Aura Biosciences is a clinical-stage biotechnology company founded in 2007 and based in Cambridge, Massachusetts. It develops cancer therapies using tumor-targeted pseudovirions, employing viral nanoparticles conjugated to infrared-activated molecules to selectively destroy cancer cells. The lead program AU-011 targets ocular melanoma, including primary choroidal melanoma, with the aim of treating tumors locally while reducing damage to surrounding tissue. The company pursues precision therapies for solid tumors and has ongoing development in other ocular oncology indications and bladder cancer, focusing operations in the United States.

Xilio Therapeutics is a clinical-stage biotechnology company focused on developing innovative immunotherapies to enhance the immune system's ability to combat cancer. The company specializes in tumor-selective immunotherapies, utilizing its proprietary technology to maximize the effectiveness of immuno-oncology treatments while minimizing side effects outside the tumor. Among its key products is XTX201, which aims to induce immune activity directly within tumors, demonstrating improved antitumor effects. Founded in 2015 and based in Waltham, Massachusetts, Xilio Therapeutics was previously known as Akrevia Therapeutics until its rebranding in March 2020. The company is dedicated to advancing a pipeline of novel immuno-oncology molecules, including tumor-activated cytokines and antibodies, with the goal of significantly improving patient outcomes in cancer treatment.

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

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Affinivax, Inc. is a biotechnology company based in Lexington, Massachusetts, specializing in the development of vaccines using its innovative Multiple Antigen Presenting System (MAPS) technology. Founded in 2014, the company aims to enhance the protection against a range of infectious diseases that affect both children and adults globally. MAPS technology allows for the effective binding of protective polysaccharides and proteins within a single vaccine, inducing robust immune responses. Affinivax is advancing vaccine programs targeting significant pathogens, including Streptococcus pneumoniae, which contributes to considerable morbidity and mortality worldwide. The company leverages insights from leading experts in infectious diseases and has secured exclusive intellectual property rights related to its MAPS platform from Boston Children’s Hospital, with notable backing from the Bill & Melinda Gates Foundation.

Biomea Fusion is a precision medicine company focused on accelerating cancer drug development by targeting specific gene alterations that drive tumor growth. Its lead program aims to disrupt the protein-to-protein interaction between menin and the MLL complex for treating various tumors.

Immuneering Corporation is a biotechnology company based in Cambridge, Massachusetts, with an additional office in New York, New York. Founded in 2008, it specializes in genetic, genomic, and proteomic data analysis for pharmaceutical clients, aiming to enhance the clinical and commercial success of medicines. The company utilizes advanced techniques such as gene expression analysis, proteomics, and next-generation sequencing to uncover biological insights that inform drug development. Immuneering's expertise in translational bioinformatics is applied throughout the drug development process, particularly for oncologic and neurologic diseases, and is integral to its proprietary computational platform, known as Disease Cancelling Technology. This platform supports drug discovery initiatives and provides computational biology services to both pharmaceutical and biotechnology companies, contributing to the creation of therapies for serious illnesses, including cancer and autoimmune diseases.

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.

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

Founded in 2019, Genesis Therapeutics is a biotechnology company based in South San Francisco, California. It specializes in developing small molecule drug candidates for treating debilitating disorders using artificial intelligence-driven platforms.

Acumen Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Livermore, California, focused on discovering and developing therapeutics and diagnostics for Alzheimer's disease and related neurodegenerative conditions. The company is advancing a targeted immunotherapy drug candidate, ACU193, which selectively targets amyloid-beta oligomers, believed to be a significant contributor to the disease process. Acumen employs a sensitivity assay to measure soluble amyloid-beta oligomer levels in cerebrospinal fluid, which aids in patient enrollment for clinical trials. The Phase 1 clinical trial for ACU193, initiated in the second quarter of 2021, aims to evaluate the drug's safety, tolerability, pharmacokinetics, and target engagement among patients with mild cognitive impairment and mild dementia due to Alzheimer's disease. Results from this trial are anticipated by the end of 2022. Founded in 1996, Acumen Pharmaceuticals is committed to developing innovative approaches to address the challenges posed by Alzheimer's disease.

Flame Biosciences is a clinical-stage biotechnology company based in New York, founded in 2017. The company specializes in developing innovative therapies for cancer and other inflammatory conditions. Its portfolio includes FL-101 therapy, designed to treat lung cancers driven by inflammation, and FL-201, aimed at reducing chronic inflammation in patients. Flame Biosciences is dedicated to improving the lives of individuals suffering from debilitating and life-threatening diseases through its transformative therapeutic approaches.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Chinook Therapeutics, Inc. is a clinical-stage biotechnology company based in Seattle, Washington, that specializes in the discovery, development, and commercialization of precision medicines for kidney diseases. The company's lead program is atrasentan, an investigational endothelin receptor antagonist currently prepared for Phase III trials targeting IgA nephropathy and other primary glomerular diseases. In addition to atrasentan, Chinook is developing BION-1301, an investigational anti-APRIL monoclonal antibody evaluated in a Phase Ib trial for IgA nephropathy, and CHK-336, a preclinical candidate aimed at treating an undisclosed ultra-orphan kidney disease. Chinook Therapeutics is also engaged in research programs addressing other rare and severe chronic kidney conditions, including polycystic kidney disease.

Cerevel Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for neuroscience diseases. Founded in 2018 and headquartered in Cambridge, Massachusetts, the company is advancing a diverse pipeline that includes five clinical-stage investigational therapies and several preclinical compounds targeting conditions such as schizophrenia, epilepsy, Parkinson's disease, and substance use disorder. Key products under development include CVL-231, a positive allosteric modulator for schizophrenia; CVL-865, which is being tested for drug-resistant focal onset epilepsy and acute anxiety; and Tavapadon, a selective dopamine D1/D5 partial agonist for Parkinson's disease. Cerevel Therapeutics employs a unique approach that leverages expertise in neurocircuitry and receptor selectivity to address unmet medical needs in the field of neuroscience.

Bolt Biotherapeutics is a biotechnology company developing cancer immunotherapies based on the Boltbody platform, which links tumor-targeting antibodies to immune-stimulating payloads to activate innate and adaptive immune responses against solid tumors. The platform aims to transform non-responsive, or cold, tumors into immunologically active ones by delivering immune stimulants at the tumor site. The company’s pipeline includes BDC-1001 as a monotherapy for HER2-expressing solid tumors, with additional candidates such as BDC-3042 and Next-Gen ISAC designed to broaden its immuno-oncology program. Bolt Biotherapeutics focuses on myeloid biology and tumor-targeted therapies to leverage immune mechanisms for cancer treatment.

Athira Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapies for brain disorders. Its lead product candidate, ATH-1017, aims to treat neurodegenerative diseases like Alzheimer's and Parkinson's by restoring neuronal health.

Everest Medicines is a clinical-stage biopharmaceutical company dedicated to developing and commercializing innovative therapies for critical unmet medical needs, initially focusing on Asia Pacific markets.

Atea Pharmaceuticals is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on discovering and developing next-generation antiviral therapeutics for severe viral diseases. The company's lead candidate, AT-527, is an antiviral agent currently undergoing phase 3 clinical trials for the treatment of COVID-19, caused by the SARS-CoV-2 virus. Additionally, Atea is advancing several candidates in clinical trials, including AT-787 for hepatitis C and AT-752 for dengue, both in phase 2 trials. The company aims to provide innovative treatments for infections caused by RNA and DNA viruses, improving outcomes for patients suffering from serious viral infections. Atea Pharmaceuticals was incorporated in 2012.

Founded in 2019 and based in Cambridge, Massachusetts, MoMa Therapeutics discovers precision medicines by targeting molecular machines underlying human diseases. The company's platform enables drug development from previously intractable enzyme classes.

Kymera Therapeutics is a biotechnology company focused on targeted protein degradation. It employs a proprietary predictive modeling platform to discover novel small molecule therapeutics that selectively degrade disease-causing proteins.

Immunocore is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

Spruce Biosciences is a biopharmaceutical company focused on developing therapies for rare endocrine disorders. Its pipeline includes tildacerfont for classic congenital adrenal hyperplasia in adults and children, with ongoing clinical development, and Tralesinidase Alfa in its portfolio. The company collaborates with Eli Lilly to research, develop, and commercialize compounds. Founded in 2014, Spruce Biosciences is headquartered in Daly City, California, and aims to deliver meaningful therapies for patients with rare endocrine diseases.

Founded in 2015, Imperative Care specializes in developing innovative medical technologies aimed at improving stroke treatment. Its flagship products include the Large Distal Platform catheter for deeper reach and the ZOOM Aspiration System for clot removal. The company's mission is to enhance treatment speed and effectiveness, ultimately optimizing patient recovery.

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

Fusion Pharmaceuticals is a clinical-stage biopharmaceutical company specializing in developing radiopharmaceuticals as precision medicines. It focuses on creating targeted alpha therapeutics using its proprietary linker technology to enhance the safety and efficacy of these drugs, ultimately improving cancer patient outcomes.

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

Vedanta Biosciences is a clinical-stage microbiome company focused on developing therapies for immune-mediated and infectious diseases using live bacteria derived from the human microbiome. The company designs orally delivered defined bacterial consortia, leveraging libraries of microbiome-derived strains, proprietary datasets from human interventional studies, and tools for selecting potent strains. Its platform encompasses consortium design and CGMP manufacturing of bacterially defined products, enabling physicians to access live bacteria drugs for autoimmune and inflammatory conditions. Headquartered in Cambridge, Massachusetts, Vedanta Biosciences aims to create a new category of oral therapies arising from defined bacterial communities grown from clonal cell banks.

Akero Therapeutics is a clinical-stage biotechnology company focused on developing treatments for serious metabolic diseases, with a primary emphasis on nonalcoholic steatohepatitis (NASH). Its lead program is a long-acting fibroblast growth factor 21 (FGF21) analog designed to restore metabolic balance and address liver fat, inflammation, and fibrosis. The company was founded in 2017 and is headquartered in South San Francisco, California.

Aprea Therapeutics, Inc. is a biopharmaceutical company based in Boston, Massachusetts, that specializes in developing cancer therapeutics aimed at reactivating the mutant p53 tumor suppressor protein. Its primary product candidate, APR-246, is a small molecule p53 reactivator currently undergoing late-stage clinical development for hematologic malignancies, particularly myelodysplastic syndromes and acute myeloid leukemia. Founded in 2006, Aprea is focused on advancing innovative treatments that target critical mechanisms in cancer biology to improve patient outcomes.

Mirum Pharmaceuticals is a biopharmaceutical company dedicated to developing and commercializing therapies for debilitating liver diseases. Its primary focus is on Maralixibat, an investigational oral drug in Phase 3 trials for treating progressive familial intrahepatic cholestasis disease and Alagille syndrome.

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

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

Kaleido Biosciences is a clinical-stage healthcare company dedicated to harnessing the potential of the microbiome to treat diseases and enhance human health. The company has developed a proprietary product platform that enables the rapid and cost-effective discovery of Microbiome Metabolic Therapies (MMTs). These therapies aim to modulate the metabolic output and profile of the microbiome by influencing the function and distribution of its existing microbes. Kaleido is advancing a diverse pipeline of MMT candidates that target various diseases and conditions, addressing significant unmet medical needs.

Innovent Biologics is a China-based biopharmaceutical company that discovers, develops, manufactures, and commercializes monoclonal antibodies. It operates a platform for antibody therapeutics across oncology, ophthalmology, immunology, and metabolic diseases, with Tyvyt (sintilimab) as its core asset, a PD-1 inhibitor used in first-line and other cancer indications. The company develops a broad pipeline including biosimilars and novel antibodies and provides distribution, consulting, and R&D services. It maintains strategic collaborations with global partners, including a co-development arrangement with MD Anderson Cancer Center and an alliance with Eli Lilly for TYVYT, and has more than 30 collaborations with major pharmaceutical companies. Innovent is listed on the Hong Kong Exchange and maintains manufacturing and development capabilities in China with a growing portfolio of oncology and non-oncology products marketed domestically and internationally.

Braeburn Inc. is a pharmaceutical company focused on developing and commercializing treatments for serious central nervous system disorders, with a primary emphasis on opioid addiction. Founded in 2012 and headquartered in Plymouth Meeting, Pennsylvania, Braeburn aims to provide innovative solutions for individuals affected by opioid use disorder (OUD). The company's lead product candidate, BRIXADI, is an extended-release injectable formulation of buprenorphine designed for the treatment of moderate to severe OUD. Braeburn is committed to advancing next-generation therapies that offer individualized dosing regimens and delivery options, striving to improve care for patients and address the growing challenges associated with addiction.

Aptinyx Inc. is a clinical-stage biopharmaceutical company based in Evanston, Illinois, that specializes in discovering and developing innovative therapies for disorders of the brain and nervous system. Founded in 2015, the company focuses on proprietary synthetic small molecules that modulate the NMDA receptor, enhancing nerve cell communication. Aptinyx's pipeline includes NYX-2925, which is in Phase II clinical development for painful diabetic peripheral neuropathy and fibromyalgia, and NYX-783, currently in Phase I trials for post-traumatic stress disorder. Additionally, NYX-458, another NMDA receptor modulator, is undergoing Phase I studies aimed at treating cognitive impairment associated with Parkinson's disease. The company also collaborates with Allergan plc to develop a compound for major depressive disorder, highlighting its commitment to addressing significant unmet medical needs in the central nervous system arena.

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

Homology Medicines is a biotechnology company focused on developing genetic therapies for rare diseases. Its proprietary platform uses human hematopoietic stem cell-derived adeno-associated virus vectors to deliver treatments in vivo, targeting various tissues including the liver and central nervous system. The company's lead product candidate, HMI-102, is in clinical trials for treating phenylketonuria.

VYNE Therapeutics Inc. is a biopharmaceutical company specializing in the development and commercialization of innovative therapies for dermatological conditions. It offers AMZEEQ, a topical minocycline treatment for non-nodular moderate-to-severe acne vulgaris in patients aged 9 and older. The company is advancing its pipeline with FMX103, currently in Phase III clinical trials for moderate-to-severe papulopustular rosacea in adults, and FCD105, a topical combination foam under investigation in Phase II trials for moderate-to-severe acne vulgaris. Additionally, VYNE is developing Serlopitant, an oral NK1 receptor antagonist aimed at addressing pruritus associated with prurigo nodularis, as well as exploring therapies for other immuno-inflammatory conditions. Formerly known as Menlo Therapeutics Inc., the company rebranded in September 2020 and is headquartered in Bridgewater, New Jersey.

Zai Lab is a biopharmaceutical company dedicated to discovering and developing transformative medicines for cancer, autoimmune, and infectious diseases. Founded in Shanghai in 2014, the company focuses on bringing innovative therapies to patients worldwide. Zai Lab's pipeline includes proprietary drug candidates such as Niraparib, Optune, Ripretinib, and Margetuximab, among others. The company collaborates with global biopharma leaders to expand its pipeline and foster innovation in China.

Synlogic, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, specializing in the development of synthetic biotic medicines aimed at treating metabolic, inflammatory, and cancer-related diseases. The company’s lead therapeutic program, SYNB1618, is currently undergoing Phase I/IIa clinical trials for the treatment of phenylketonuria, a rare genetic metabolic disorder. Additionally, Synlogic is advancing SYNB1891, an intratumoral synthetic biotic medicine in Phase I clinical trials targeting solid tumors and lymphoma. The company employs a proprietary approach that utilizes engineered probiotics, specifically Escherichia coli Nissle 1917, to create GI-restricted oral therapies that are non-colonizing and reversible, facilitating easier administration and storage. Synlogic is also exploring treatments for other conditions, including secondary hyperoxaluria, inflammatory bowel disease, and various metabolic disorders, and has established a collaboration with Ginkgo Bioworks to further its development efforts.

Spero Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing novel treatments for multi-drug resistant bacterial infections and rare diseases. Its primary focus is on tebipenem pivoxil hydrobromide, an oral carbapenem-class antibiotic intended for adult use in treating MDR Gram-negative infections.

Bristol-Myers Squibb is a global biopharmaceutical company that discovers, develops, manufactures, and markets medicines across hematology, oncology, cardiovascular, and immunology. Its portfolio includes Opdivo for cancer immunotherapy; Eliquis for stroke prevention and venous thromboembolism; Orencia for rheumatoid arthritis; Sprycel, Yervoy, Empliciti, Baraclude; Reyataz and Sustiva for HIV; and Daklinza, Sunvepra, Beclabuvir for viral infections. The company sells products through wholesalers, retail pharmacies, hospitals, government entities, and medical professionals worldwide, and pursues collaboration and licensing arrangements with Pfizer, Otsuka, Ono, Nektar, AstraZeneca, Insitro, Schrödinger, and others. It also engages in partnerships for discovery and development with multiple biotech firms and academia. Founded in 1887 and headquartered in New York, the firm has a strong presence in immuno-oncology and focuses on innovative therapies and global reach.

Biohaven Pharmaceuticals is a clinical-stage biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative treatments for neurological diseases and other therapeutic areas. The company focuses on life-changing therapies targeting conditions such as neuromuscular disorders, metabolic diseases, obsessive-compulsive disorder, and various forms of cancer. Its diverse pipeline includes late-stage product candidates that utilize mechanisms such as calcitonin gene-related peptide receptor antagonists, glutamate modulators, and myeloperoxidase inhibitors. Notable candidates in its pipeline include BHV3000-301, BHV3000-302, and BHV3000-303, among others. Biohaven's commitment to addressing unmet medical needs positions it as a significant player in the biopharmaceutical landscape.

Ardelyx is a biopharmaceutical company developing first-in-class, minimally absorbed oral medicines to treat mineral metabolism and metabolic disorders. Based in Fremont, California, founded in 2007. It focuses on non-systemic therapies that act in the intestines to modulate transporters and receptors, aiming to limit systemic exposure. Its lead product Tenapanor targets irritable bowel syndrome with constipation and hyperphosphatemia in chronic kidney disease on dialysis; the program includes Phase 3 trials. The company is pursuing additional candidates such as RDX013 for hyperkalemia, and RDX002 and RDX009 in earlier stages. Ardelyx aims to address cardiorenal and metabolic conditions using a non-systemic platform.

Mersana Therapeutics is a clinical-stage biopharmaceutical company that develops antibody-drug conjugates (ADCs) for cancer patients with unmet medical needs. It leverages proprietary ADC platforms, including Dolaflexin, Dolasynthen, and Immunosynthen, to create a diverse pipeline of product candidates designed to improve therapeutic benefit and address oncology indications underserved by traditional ADCs. The lead candidate XMT-1536 targets NaPi2b and is in Phase I trials for ovarian cancer, non-small cell lung cancer, and other indications. The company also advances additional programs such as Emi-Le (XMT-1660), XMT-2056, XMT-2068, and XMT-2175, reflecting its focus on next-generation ADCs. Mersana is headquartered in Cambridge, Massachusetts, and was founded in 2005.

Vedanta Biosciences is a clinical-stage microbiome company focused on developing therapies for immune-mediated and infectious diseases using live bacteria derived from the human microbiome. The company designs orally delivered defined bacterial consortia, leveraging libraries of microbiome-derived strains, proprietary datasets from human interventional studies, and tools for selecting potent strains. Its platform encompasses consortium design and CGMP manufacturing of bacterially defined products, enabling physicians to access live bacteria drugs for autoimmune and inflammatory conditions. Headquartered in Cambridge, Massachusetts, Vedanta Biosciences aims to create a new category of oral therapies arising from defined bacterial communities grown from clonal cell banks.

G1 Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Research Triangle Park, North Carolina, focused on the discovery, development, and commercialization of small molecule therapeutics for cancer treatment. The company is advancing several key drug candidates, including trilaciclib, an intravenous cyclin-dependent kinases (CDK) 4/6 inhibitor currently in Phase 1b/2 clinical trials for extensive-stage small cell lung cancer and Phase 2 trials for first-line small cell lung cancer and metastatic triple-negative breast cancer. Additionally, G1 is developing lerociclib, an oral CDK4/6 inhibitor in Phase 1b/2a trials for breast cancer and Phase 1b trials for non-small cell lung cancer, as well as rintodestrant, an oral selective estrogen receptor degrader in Phase I/2 trials. The company collaborates with Quantum Leap Healthcare Collaborative to explore trilaciclib's potential in neoadjuvant treatment for locally advanced breast cancer. Founded in 2008, G1 Therapeutics aims to address unmet medical needs in oncology by enhancing the effectiveness of existing cancer therapies.

Clearside Biomedical is a biopharmaceutical company focused on developing treatments for serious eye diseases. It specializes in delivering drugs directly to specific compartments of the eye using its proprietary suprachoroidal injection platform, aiming to restore and preserve vision.

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 affecting women. Its key products in development include Linzagolix, an oral gonadotropin-releasing hormone receptor antagonist aimed at treating pain from endometriosis and heavy menstrual bleeding associated with uterine fibroids. ObsEva is also working on OBE022, a once-daily oral prostaglandin F2α receptor antagonist designed for managing preterm labor between 24 and 34 weeks of gestation, and 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 within the Swiss market while targeting global solutions for women's reproductive health needs.

Audentes Therapeutics, a clinical stage biotechnology company based in San Francisco, specializes in developing gene therapy products aimed at treating serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapeutic candidates, including AT132 for X-linked myotubular myopathy, AT342 for Crigler-Najjar syndrome, and AT845, which is in preclinical studies for Pompe disease. Additionally, Audentes is developing vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. It has established collaborative agreements with Genethon and the University of Pennsylvania to further research and commercialization efforts for these therapies. Founded in 2012, Audentes Therapeutics was acquired by Astellas Pharma in January 2020 and operates under the name Astellas Gene Therapies, focusing on gene therapy research, development, and future commercialization.

Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company focused on immuno-oncology therapies that modulate the immune system to treat cancer. Its pipeline includes Ciforadenant, an oral A2A receptor antagonist, CPI-006, an anti-CD73 antibody, CPI-818, an ITK inhibitor, and soquelitinib, an ITK-targeting agent. The company was founded in 2014 and is based in Burlingame, California.

Aclaris Therapeutics is a United States-based clinical-stage biopharmaceutical company focused on developing novel therapies for immuno-inflammatory diseases and dermatology, aiming to address unmet medical and aesthetic needs where treatment options are limited. The company operates two segments, therapeutics and contract research, with the therapeutics business pursuing innovative treatments for immuno-inflammatory conditions while the contract research segment provides laboratory services. Its pipeline includes Zunsemetinib, an oral MK2 inhibitor, and ATI-2138, among other drug candidates.

Proteostasis Therapeutics is a clinical-stage biopharmaceutical company focused on developing small molecule therapies that regulate the body's protein homeostasis network to treat cystic fibrosis and other genetic or degenerative diseases. It develops Proteostasis Regulators intended to restore proteostasis balance and address conditions such as cystic fibrosis, emphysema, diabetes, and neurodegenerative disorders. In CF, its lead candidates include PTI-801, a CFTR corrector; PTI-808, a CFTR potentiator; and PTI-428, a CFTR amplifier. The company collaborates with the Cystic Fibrosis Foundation to research, develop, and commercialize CF-related products, and has licensing relationships with Genentech. It was founded in 2006, formerly known as Proteoguard, and was renamed Proteostasis Therapeutics in 2007, based in Boston. It was acquired by Yumanity Therapeutics in 2020 in a reverse merger.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.