RTW Investments

AIRNA is a biotech company that is pioneering RNA editing therapies to improve the health of individuals suffering from both rare and common ailments.

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

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

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

Santa Ana Bio is a biotechnology company focused on developing precision therapies for autoimmune and inflammatory diseases. By utilizing advanced technologies such as deep cell profiling, single-cell genomics, and multi-omics platforms, the company designs targeted biologics that aim to provide effective and personalized treatment options. Through its innovative approach, Santa Ana Bio seeks to enhance patient outcomes and improve quality of life for those affected by autoimmune conditions.

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.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

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

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company has a diverse pipeline that includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. A key aspect of their development efforts includes a small molecule cardiac myosin inhibitor, in-licensed from Cytokinetics, specifically designed to treat hypertrophic cardiomyopathy. Jixing Pharmaceuticals aims to provide effective treatments for patients in China, addressing significant unmet medical needs in the realms of cardiovascular and ophthalmic health.

SAB Biotherapeutics, Inc. is a clinical-stage biopharmaceutical development company based in Sioux Falls, South Dakota, with an additional office in Cambridge, Massachusetts. Founded in 2014, the company focuses on developing immunotherapies for various human diseases using its proprietary DiversitAb platform. This innovative platform enables the rapid production of fully targeted human polyclonal antibodies from genetically engineered transchromosomic cattle, eliminating the need for human plasma or serum. SAB Biotherapeutics' product pipeline includes SAB-142 for Type 1 diabetes, SAB-176 for seasonal influenza, and SAB-301 for Middle East Respiratory Syndrome Coronavirus (MERS-CoV). The company's approach aims to address public health challenges, rare diseases, and pandemic threats through advanced antibody science.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Kyverna Therapeutics, Inc. is a cell therapy company focused on developing innovative treatments and potential cures for serious autoimmune diseases. Founded in 2018 and based in Berkeley, California, Kyverna employs advanced T cell engineering and synthetic biology technologies to target and eliminate autoreactive immune cells, which are responsible for the inflammation associated with these diseases. By addressing the root causes of autoimmune conditions, Kyverna aims to provide more effective and lasting solutions for patients suffering from these challenging health issues.

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.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

MoonLake Immunotherapeutics is a clinical-stage biopharmaceutical company crating novel therapies, and unlocking the potential of Sonelokimab, the novel investigational Nanobody® for the treatment of inflammation, to revolutionize outcomes for patients.

22seventy bio is an immuno-oncology cell therapy company that discovers and develops cancer therapies. The company combines a deep knowledge of cancer cell biology and genetics with an understanding of the immune response to cancer to advance its pipeline. It was established in Cambridge, Massachusetts in 2021.

Kyverna Therapeutics, Inc. is a cell therapy company focused on developing innovative treatments and potential cures for serious autoimmune diseases. Founded in 2018 and based in Berkeley, California, Kyverna employs advanced T cell engineering and synthetic biology technologies to target and eliminate autoreactive immune cells, which are responsible for the inflammation associated with these diseases. By addressing the root causes of autoimmune conditions, Kyverna aims to provide more effective and lasting solutions for patients suffering from these challenging health issues.

Lycia Therapeutics is a biotechnology firm focused on discovering and developing first-in-class therapies. The company utilizes its innovative lysosomal targeting chimeras (LYTACs) platform to create therapeutics that target and degrade extracellular and membrane-bound proteins associated with challenging diseases, including cancer and autoimmune disorders. By addressing these difficult-to-treat conditions, Lycia Therapeutics aims to provide medical practitioners with effective treatment options to improve patient outcomes.

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

Renovacor, Inc. is a preclinical-stage biopharmaceutical company based in Philadelphia, Pennsylvania, dedicated to developing gene therapies for cardiovascular diseases. Founded in 2013, the company focuses on innovative treatments for dilated cardiomyopathy (DCM), particularly cases caused by mutations in the BAG3 gene. This condition affects over 3 million individuals in the United States, with around 35,000 patients estimated to have BAG3 mutations, classifying it as an orphan disease. Patients with BAG3-associated DCM, often younger and experiencing faster disease progression, currently have limited treatment options, primarily consisting of standard heart failure care. Renovacor's lead program involves a recombinant adeno-associated virus (AAV)-based gene therapy aimed at replacing the defective BAG3 gene, which holds the potential to prevent disease progression and improve outcomes for this vulnerable patient population.

Artios Pharma Limited is a biotechnology company based in Cambridge, United Kingdom, that specializes in developing innovative cancer treatments targeting DNA damage response (DDR) pathways. Founded in 2015, the company is dedicated to creating first-in-class therapies aimed at selectively killing cancer cells. Artios's product pipeline features several promising programs, including a focus on DNA polymerase theta (Polθ), which plays a crucial role in DNA repair processes. Additionally, the company is advancing an in-licensed program that targets a newly identified protein linked to DDR and is developing a novel treatment aimed at DNA nucleases involved in these pathways. Artios collaborates with Cancer Research Technology and prominent DNA repair researchers globally to enhance its research and development efforts.

Jixing Pharmaceuticals is a global biotechnology company focused on developing and commercializing innovative therapeutics for cardiovascular and ophthalmic diseases. The company has a diverse pipeline that includes treatments for conditions such as hypertrophic cardiomyopathy, heart failure, presbyopia, hypertension, and dry eye disease. A key aspect of their development efforts includes a small molecule cardiac myosin inhibitor, in-licensed from Cytokinetics, specifically designed to treat hypertrophic cardiomyopathy. Jixing Pharmaceuticals aims to provide effective treatments for patients in China, addressing significant unmet medical needs in the realms of cardiovascular and ophthalmic health.

Umoja Biopharma specializes in innovative immunotherapy aimed at transforming cancer treatment and enhancing patient quality of life. The company focuses on reprogramming T cells within the patient's body to effectively target cancer, including solid tumors and hematological cancers that often have poor responses to conventional therapies. Its proprietary CAR T-cell gene therapy platform is designed to deliver scalable therapeutic regimens tailored to various stages of cancer, enabling medical practitioners to directly and safely attack cancer cells. By harnessing the body's immune response, Umoja Biopharma seeks to improve overall patient outcomes and provide more effective treatment options for those affected by cancer.

Pyxis Oncology is a developer of antibody therapeutics intended to promote the body's immune response to cancer. The company has engaged in deep analyses of tumor antigen-specific tumor-infiltrating lymphocytes (TILs) within hot tumors and of tumor cell signaling pathways within cold tumors, providing laboratories with development of novel antibody-based immunotherapies. It was founded in 2019 and is based in Boston, Massachusetts.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Tenaya Therapeutics is a developer of novel therapies designed to offer treatment for heart disease. The company's therapies address heart failure through multipronged efforts that target the fundamental cellular pathologies present in diseased cardiac muscle and that leverage cutting-edge research in cardiac development and regeneration, enabling physicians to regenerate heart tissue, and additional programs focused on cardiomyopathies.

Artiva Biotherapeutics is a biotech company focused on developing and manufacturing cellular immunotherapies for cancer patients. Founded in 2019 and based in San Diego, California, Artiva specializes in off-the-shelf, allogeneic natural killer (NK) cell therapies aimed at treating various hematologic cancers and solid tumors. The company's product pipeline targets specific antigens, including CD20 and CD19 for B-cell lymphomas, as well as HER2 for certain solid tumors. Artiva's approach emphasizes the therapeutic potential of NK cells and aims to overcome challenges in scaling and manufacturing these therapies. The company is committed to delivering safe, effective, and readily accessible cell therapies to benefit cancer patients. Artiva operates as a subsidiary of Green Cross Holdings Corporation.

Encoded Therapeutics, Inc. is a biotechnology company that specializes in precision gene therapies aimed at treating a wide array of severe genetic disorders. Utilizing a unique platform, Encoded identifies sequences within the human genome that regulate gene expression through advanced screening and computational techniques. The company's therapy pipeline targets both genetic and acquired disorders across various disease pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and based in South San Francisco, California, Encoded Therapeutics is focused on developing innovative solutions that enable medical practitioners to improve patient outcomes and enhance quality of life.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Cogent Biosciences is a biotechnology company focused on developing precision therapies for genetically defined diseases, utilizing proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body’s immune system in the fight against cancer. The company designs targeted treatments aimed at addressing the underlying causes of diseases, thereby improving patient outcomes. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor that specifically targets the KIT D816V mutation and other mutations in KIT exon 17. This mutation is predominantly linked to Systemic Mastocytosis, a severe condition resulting from the uncontrolled growth of mast cells, and is also associated with advanced gastrointestinal stromal tumors. Cogent Biosciences is advancing its lead program, which is expected to enter Phase I clinical trials to evaluate safety and efficacy.

C4 Therapeutics, Inc. is a biopharmaceutical company focused on developing innovative therapeutic candidates that target and eliminate disease-causing proteins, primarily for the treatment of cancer and neurodegenerative disorders. The company's lead product candidate, CFT7455, is an orally bioavailable degrader designed to target IKZF1/3 for multiple myeloma and other lymphomas. Additionally, C4 is advancing CFT8634, which targets BRD9 for specific solid tumors, alongside programs aimed at genetically defined resistant solid tumors. C4 Therapeutics utilizes its proprietary Degronimid platform, which allows for the selective degradation of proteins through the natural ubiquitin/proteasome system, potentially addressing previously undruggable targets while minimizing drug resistance. The company has formed strategic collaborations with major industry players, and it aims to make a significant impact on the treatment landscape through its advanced protein degradation science and multiple development programs. Founded in 2015 and headquartered in Watertown, Massachusetts, C4 Therapeutics continues to innovate in the field of targeted therapy.

Athira Pharma, Inc. is a clinical-stage biopharmaceutical company based in Seattle, Washington, focused on developing small molecules aimed at restoring neuronal health and addressing neurodegenerative diseases. The company's lead candidate, ATH-1017, is a small hepatocyte growth factor/MET activator designed to penetrate the blood-brain barrier and is currently undergoing various clinical trials for the treatment of Alzheimer's and Parkinson's diseases. Additionally, Athira is advancing other candidates, including ATH-1019, which is in preclinical development for depression, and ATH-1018, aimed at treating peripheral neuropathy. Founded in 2011 and originally named M3 Biotechnology, Athira Pharma changed its name in April 2019 to reflect its commitment to innovative therapies that positively impact individuals affected by brain disorders.

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.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

Avidity Biosciences, Inc. is a biotechnology company focused on developing oligonucleotide-based therapies known as antibody oligonucleotide conjugates (AOCs). These innovative therapies aim to address the limitations of traditional oligonucleotide treatments by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide therapies. This approach allows Avidity to target previously undruggable tissue and cell types, thereby addressing the underlying genetic drivers of various diseases. The company's lead product candidate, AOC 1001, is designed to treat myotonic dystrophy type 1, a rare muscle disease. Additionally, Avidity is advancing multiple programs aimed at treating muscle-related conditions such as muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Founded in 2012 and based in La Jolla, California, Avidity is also exploring therapeutic applications in immune and other cell types.

Frequency Therapeutics is a clinical-stage biotechnology company based in Woburn, Massachusetts, that aims to repair or reverse damage from various degenerative diseases by utilizing the body’s own biological mechanisms. The company employs a Progenitor Cell Activation approach, which involves the use of small molecules to activate progenitor cells and promote the regeneration of functional tissue. Its leading product candidate, FX-322, is currently undergoing phase 2a clinical trials to address the underlying causes of sensorineural hearing loss. In addition to hearing loss, Frequency Therapeutics is also exploring treatments for a range of other conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. The company has established a collaboration with Astellas Pharma for the development and commercialization of FX-322. Founded in 2014, Frequency Therapeutics focuses on disease modification without the complexities associated with genetic engineering.

Encoded Therapeutics, Inc. is a biotechnology company that specializes in precision gene therapies aimed at treating a wide array of severe genetic disorders. Utilizing a unique platform, Encoded identifies sequences within the human genome that regulate gene expression through advanced screening and computational techniques. The company's therapy pipeline targets both genetic and acquired disorders across various disease pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and based in South San Francisco, California, Encoded Therapeutics is focused on developing innovative solutions that enable medical practitioners to improve patient outcomes and enhance quality of life.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

Orchard Therapeutics is a biopharmaceutical company based in the United Kingdom that specializes in developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which modifies a patient's own hematopoietic stem cells to create a targeted treatment administered in a single dose. Orchard's portfolio includes Strimvelis, the first autologous gene therapy approved by the European Medicines Agency for the treatment of adenosine deaminase-severe combined immunodeficiency (ADA-SCID). Additionally, it is advancing clinical programs for various conditions, including metachromatic leukodystrophy, Wiskott-Aldrich syndrome, X-linked chronic granulomatous disease, and transfusion-dependent beta-thalassemia. The company also has an extensive preclinical pipeline targeting mucopolysaccharidosis types I, IIIA, and IIIB. Orchard Therapeutics collaborates with leading institutions in gene therapy research, enhancing its efforts to transform the lives of patients with rare diseases in the UK, European Union, and the United States. Founded in 2015 and headquartered in London, it aims to provide sustainable clinical benefits through its innovative therapies.

ARMO BioSciences, Inc. is an immuno-oncology company based in Redwood City, California, focused on developing biologic therapeutics that stimulate the immune system to combat cancer. The company's product pipeline includes several innovative candidates, such as AM0010, a long-acting form of interleukin-10 that enhances immune response, and AM0001, an anti-PD-1 monoclonal antibody targeting various cancers. Other key products in development include an anti-LAG-3 checkpoint inhibitor, AM0015, a recombinant form of interleukin-15, and AM0012, a recombinant interleukin-12. Established in 2010 and formerly known as Targenics, ARMO BioSciences aims to activate and enhance the body's immune response against tumors, thereby improving outcomes for cancer patients. As of mid-2018, it operates as a subsidiary of Eli Lilly and Company.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a severe genetic muscle-wasting disease primarily affecting boys. The company's lead product candidate, SGT-001, is a gene transfer therapy currently undergoing a Phase I/II clinical trial aimed at restoring functional dystrophin protein expression in patients’ muscles. DMD is a progressive and ultimately fatal condition, with an estimated prevalence of 10,000 to 15,000 cases in the United States. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which targets the LTBP4 protein to reduce fibrosis and inflammation. The company also develops biomarkers, sensors, and the Solid Suit program, which includes wearable assistive devices designed to provide functional and therapeutic benefits. Established in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences collaborates with Ultragenyx to enhance its gene therapy initiatives for DMD.

Immunocore Limited is a biotechnology company based in Abingdon, United Kingdom, with a focus on discovering and developing novel therapeutics for cancer, viral diseases, and autoimmune conditions. The company specializes in T cell receptor (TCR) technology, which allows it to create innovative bispecific immunotherapies. Its lead product candidate, IMCgp100, is currently undergoing clinical trials for the treatment of metastatic melanoma. Immunocore's proprietary platform enables efficient manufacturing of TCR-based drugs, addressing significant unmet medical needs. Founded in 1999 and previously known as Avidex Limited, Immunocore has expanded its operations with an additional office in Conshohocken.

REGENXBIO Inc. is a clinical-stage biotechnology company specializing in gene therapy aimed at addressing genetic defects and enabling cells to produce therapeutic proteins and antibodies. The company utilizes its proprietary NAV Technology Platform, which involves recombinant adeno-associated virus vectors for gene delivery. REGENXBIO’s lead candidate, RGX-314, is currently in Phase I/IIa clinical trials for the treatment of wet age-related macular degeneration. Additional product candidates under development include RGX-121 for mucopolysaccharidosis type II, RGX-111 for mucopolysaccharidosis type I, RGX-181 for late infantile neuronal ceroid lipofuscinosis type II, and RGX-501 for homozygous familial hypercholesterolemia. Beyond its own product candidates, REGENXBIO also licenses its NAV Technology Platform to other biotechnology and pharmaceutical companies and has a collaboration agreement with Neurimmune AG to develop vectorized antibodies for neurodegenerative diseases. Founded in 2008 and headquartered in Rockville, Maryland, the company was previously known as ReGenX Biosciences, LLC before rebranding in 2014.