Rock Springs Capital

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.

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.

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.

Crinetics Pharmaceuticals is a clinical-stage company focused on discovering, developing, and commercializing therapeutics targeting peptide hormone receptors for treating rare endocrine diseases and related cancers. Its lead product candidate, paltusotine, is an oral selective nonpeptide somatostatin receptor type 2 biased agonist in Phase I trials for acromegaly and neuroendocrine tumors.

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.

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.

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.

immatics is a biopharmaceutical company focused on the discovery of true targets for cancer immunotherapies with the development of the right T cell receptors. The company's customized Adoptive Cell Therapies (ACTengine) and antibody-like TCR Bispecifics are the two main product classes in the pipeline (TCER). With a focus on particularly difficult-to-treat solid malignancies, each therapy modality has unique characteristics to create the desired therapeutic impact for patients at various disease stages and with various types of tumors. immatics biotechnologies was established in 2000 by Hans Rammensee, Harpreet Singh, and Steffen Walter in Tübingen, Baden-Wurttemberg.

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.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

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.

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.

Freenome, Inc. is an artificial intelligence genomics biotech company based in South San Francisco, California, founded in 2014. The company focuses on developing accurate, accessible, and non-invasive blood tests for the early detection of cancer and treatment selection. Utilizing a proprietary multi-omics platform, Freenome employs advanced algorithms to analyze cell-free genomic data, which aids in identifying early-stage cancers and other diseases. This innovative approach aims to transform disease management by enabling timely interventions and proactive treatment strategies. In addition to its diagnostic solutions, Freenome also offers clinical research services to further advance cancer detection and management.

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.

Disc Medicine is a clinical-stage biopharmaceutical company focused on developing innovative treatments for patients with severe hematologic disorders. Its pipeline includes therapies targeting erythropoietic porphyrias, Diamond-Blackfan Anemia, anemia of myelofibrosis and chronic kidney disease, as well as polycythemia vera.

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.

dMed Company Limited, also known as dMed Biopharmaceutical Co., Ltd., is a clinical contract research organization based in Shanghai, China. Established in 2016, dMed specializes in offering a comprehensive range of services to support the development of medicines and medical devices. Its offerings include consulting, clinical operations, biostatistics and programming, data management, pharmacovigilance, clinical science and medical affairs, quality assurance, and regulatory affairs and strategy. The company serves a diverse clientele, including biopharmaceutical and medical device companies, and aims to establish itself as a prominent international brand in the field of drug innovation services and solutions.

Clinipace Inc. is a clinical research organization based in Morrisville, North Carolina, with additional offices in Boulder, Colorado, Buenos Aires, Argentina, São Paulo, Brazil, and various locations in Europe and Asia Pacific. Founded in 2003, the company provides fully integrated clinical research services tailored for venture-backed, mid-tier, and strategic pharmaceutical, biotechnology, and medical device companies both in the United States and internationally. Clinipace specializes in a range of therapeutic areas, including oncology, nephrology, gastroenterology, cardiology, and immunology, among others. Its comprehensive suite of services encompasses project management, clinical monitoring, biostatistics, patient recruitment, regulatory affairs, and legal representation, allowing for a collaborative and flexible approach to clinical research that distinguishes it from traditional contract research organizations.

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.

BioTheryX is a clinical-stage biopharmaceutical company focused on restoring protein homeostasis through targeted protein degradation. It specializes in developing innovative small molecule degraders, initially targeting cancer, using its proprietary PRODEGY platform built upon expertise in Cereblon binding.

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.

Graphite Bio is a clinical-stage gene editing company focused on targeted DNA integration to treat serious diseases. Its platform aims to precisely insert, repair, or replace genetic sequences by integrating therapeutic cargoes at predefined genomic locations, enabling correction of mutations or replacement of disease-causing genes while preserving normal regulatory control. Based in South San Francisco, the company pursues high-efficiency precision gene editing to address underlying causes of disease.

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.

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

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.

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.

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.

Freenome, Inc. is an artificial intelligence genomics biotech company based in South San Francisco, California, founded in 2014. The company focuses on developing accurate, accessible, and non-invasive blood tests for the early detection of cancer and treatment selection. Utilizing a proprietary multi-omics platform, Freenome employs advanced algorithms to analyze cell-free genomic data, which aids in identifying early-stage cancers and other diseases. This innovative approach aims to transform disease management by enabling timely interventions and proactive treatment strategies. In addition to its diagnostic solutions, Freenome also offers clinical research services to further advance cancer detection and management.

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.

New Horizon Health is a biotechnology company based in Hangzhou, China, with an additional office in Beijing, specializing in the development of innovative technologies for early cancer detection and screening. Founded in 2015, the company primarily focuses on gastrointestinal cancers, offering products such as the ColoClear and Pupu Tube, which utilize fecal gene analysis (FIT-DNA) technology for colorectal cancer screening. These products enable users to conduct in-home tests, allowing for the early detection of colon cancer and precancerous lesions that may not be identified through traditional blood tests. New Horizon Health aims to reduce cancer morbidity and mortality rates by promoting accessible and efficient health services, leveraging big data and artificial intelligence in its screening technologies. The company's commitment to early cancer screening reflects its mission to enhance public health by facilitating widespread access to vital diagnostic tools.

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.

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.

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.

resTORbio, Inc., founded in 2016 and headquartered in Boston, Massachusetts, was a clinical-stage biopharmaceutical company focused on developing medicines to treat aging-related diseases. Its primary approach involved selectively inhibiting the target of rapamycin complex 1 (TORC1), a pathway linked to age-related decline in organ functions. The company's lead drug candidate, RTB101, an oral and potent TORC1 inhibitor, was being evaluated in Phase 1b/2a clinical trials for Parkinson's disease and other neurodegenerative diseases. Prior to its acquisition by Adicet Bio in September 2020, resTORbio collaborated with TrialSpark to investigate RTB-101 as a potential treatment for COVID-19.

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.

AveXis, operating as Novartis Gene Therapies, Inc., is a biotechnology company focused on developing and commercializing gene therapies for rare and life-threatening neurological genetic diseases. Acquired by Novartis in 2018, the company specializes in innovative treatments such as motor neuron cell-targeted gene replacement therapy aimed at conditions like spinal muscular atrophy, Rett syndrome, and amyotrophic lateral sclerosis. These therapies are designed to assist healthcare institutions in addressing diseases that cause progressive muscle weakness, paralysis, and ultimately, death. Through its advancements in gene therapy, AveXis is dedicated to improving the lives of patients and families impacted by these severe genetic disorders.