RTW Investments

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.

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.

LENZ Therapeutics is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative ophthalmic therapies aimed at improving vision. The company's primary focus is on addressing presbyopia, a common condition that leads to the gradual loss of near vision, affecting nearly all individuals over the age of 45. LENZ Therapeutics is currently advancing an aceclidine-based eye drop specifically designed to restore near vision, addressing a significant unmet need in the market. Through its efforts, the company aims to enhance the quality of life for individuals experiencing vision changes related to aging.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). This chronic condition, which encompasses disorders such as ulcerative colitis and Crohn's disease, involves inflammation in the gastrointestinal tract. Spyre Therapeutics combines advanced antibody engineering with rational therapeutic combinations and precision medicine strategies to enhance patient selection. The company's goal is to create next-generation products that improve upon existing treatment options, which include anti-inflammatory drugs, immunosuppressants, and biologics. Through its robust pipeline, Spyre Therapeutics seeks to address the unmet needs of patients suffering from IBD.

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.

Basking Biosciences is a startup focused on developing a reversible thrombolytic therapy aimed at restoring blood flow to the brain during ischemic strokes, thereby preventing hemorrhage and minimizing long-term damage. The company’s innovative approach involves a paired therapy that combines an active agent targeting a crucial component of clot stability with a designed reversal agent. This reversal agent can be administered if bleeding occurs, effectively neutralizing the active compound and restoring the blood's normal clotting ability. By enabling healthcare professionals to treat acute ischemic stroke effectively, Basking Biosciences aims to improve patient outcomes in critical medical situations.

Dianthus Therapeutics is a clinical-stage biotechnology company focused on developing innovative treatments for severe autoimmune diseases through targeted therapies. The company's lead candidate, DNTH103, is a monoclonal antibody designed to selectively inhibit a specific component of the immune system, offering potential advantages over existing complement therapies. This treatment aims to improve safety and efficacy for patients suffering from serious autoimmune and inflammatory conditions. Dianthus is committed to advancing its pipeline of next-generation complement therapeutics, supported by a team of experienced professionals in the biotech and pharmaceutical sectors. The company envisions that DNTH103 could be administered as a self-injection for certain patients, enhancing convenience and accessibility in treatment.

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.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies for chronic inflammatory and autoimmune diseases. The company has a pipeline that includes three small molecule products. Its lead program, IMU-838, is a selective immune modulator that works by inhibiting the enzyme DHODH, targeting conditions such as multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. The second product, IMU-935, functions as an inverse agonist of the transcription factor RORγt and is being developed for psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Lastly, IMU-856 aims to restore intestinal barrier function and is intended for diseases associated with bowel barrier dysfunction. Through these innovative therapies, Immunic Therapeutics aims to address significant unmet medical needs in the field of immunology.

Spyre Therapeutics is a biotechnology company focused on developing innovative antibody therapies aimed at transforming the treatment landscape for inflammatory bowel disease (IBD). This chronic condition, which encompasses disorders such as ulcerative colitis and Crohn's disease, involves inflammation in the gastrointestinal tract. Spyre Therapeutics combines advanced antibody engineering with rational therapeutic combinations and precision medicine strategies to enhance patient selection. The company's goal is to create next-generation products that improve upon existing treatment options, which include anti-inflammatory drugs, immunosuppressants, and biologics. Through its robust pipeline, Spyre Therapeutics seeks to address the unmet needs of patients suffering from IBD.

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.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

OriCell Therapeutics develop drugs with good efficacy and affordable prices to satisfy the globally unmet clinical needs through innovations and strives to become the world-leading developer of novel drugs for tumor immunotherapy.

Immunic Therapeutics is a clinical-stage biopharmaceutical company focused on developing selective oral immunology therapies for chronic inflammatory and autoimmune diseases. The company has a pipeline that includes three small molecule products. Its lead program, IMU-838, is a selective immune modulator that works by inhibiting the enzyme DHODH, targeting conditions such as multiple sclerosis, ulcerative colitis, Crohn's disease, and primary sclerosing cholangitis. The second product, IMU-935, functions as an inverse agonist of the transcription factor RORγt and is being developed for psoriasis, castration-resistant prostate cancer, and Guillain-Barré syndrome. Lastly, IMU-856 aims to restore intestinal barrier function and is intended for diseases associated with bowel barrier dysfunction. Through these innovative therapies, Immunic Therapeutics aims to address significant unmet medical needs in the field of immunology.

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.

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

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.

Magnolia Medical Technologies, Inc. is a Seattle-based company that specializes in developing medical devices aimed at enhancing the accuracy of diagnostic blood culture tests. The company's flagship product, SteriPath, is a vacuum-assisted blood collection system designed to eliminate contamination from initial blood samples, thereby reducing false-positive results that can lead to misdiagnosis of conditions like sepsis. This innovative system employs a pre-assembled and sterile collection method that actively diverts potentially contaminated blood, allowing for the collection of uncontaminated samples through a separate pathway. By improving the quality of diagnostic specimens, Magnolia Medical Technologies seeks to enhance patient outcomes, lower hospital costs, and address the growing issue of antibiotic resistance. The company, incorporated in 2011, also holds a substantial intellectual property portfolio, which includes over 70 issued patents and more than 50 additional patent applications pending.

Orchestra BioMed, Inc. is a biomedical innovation company focused on developing high impact, high value, evidence-based therapeutic solutions for medical procedures that address major medical conditions. Its main focus is on developing new therapies for cardiovascular diseases which are the leading cause of death worldwide, taking the lives of 17.7 million people each year, or 31% of all global deaths according to the World Health Organization. It was founded in 2017 and is headquartered in New Hope, Pennsylvania.

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.

Third Harmonic Bio is a clinical-stage company developing a first-in-class, highly selective, oral KIT inhibitor for treatment of severe allergy and inflammation. KIT is a cell surface receptor that acts as the master survival and functional regulator of mast cells, which are immune cells concentrated at the body’s interfaces with the external environment and that act as the key mediator of the inflammation associated with allergic diseases.

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

CinCor Pharma, Inc. is a clinical-stage biopharmaceutical company based in Cincinnati, Ohio, established in 2018. The company is dedicated to developing clinical candidates targeting cardiovascular, metabolic, and kidney diseases. Its primary focus is on CIN-107, an aldosterone synthase inhibitor aimed at treating resistant hypertension and primary aldosteronism. CinCor Pharma's mission is to advance its promising clinical candidates toward marketing approval, emphasizing the development of innovative therapies for patients suffering from cardio-renal conditions.

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.

Alcyone Therapeutics is Pioneering Next-Generation Precision CNS Genetic Therapies to unlock new possibilities and overcome challenges for CNS therapy development.

NiKang Therapeutics is an early-stage biotech company focused on discovering and developing innovative small molecule oncology medicines to help patients with unmet medical needs. Its discovery approach is informed by target structure biology and capitalizes on structure-based drug design enabling a rapid and efficient discovery and development of proprietary drug candidates with the most desirable pharmacological features into clinical studies.

Numab is a biotechnology company focused on developing antibody-based therapeutics for severe diseases, including chronic inflammation and cancer. The company employs a plug-and-play platform that minimizes the randomness typically associated with the drug discovery process, allowing for the predictable creation of multispecific biotherapeutics. This platform facilitates the development of mono- or multispecific antibody fragment-based therapeutics with customized pharmacokinetic properties, enabling the healthcare sector to address multiple therapeutic targets simultaneously. Through its innovative approach, Numab aims to enhance the effectiveness of immunotherapies in treating complex medical conditions.

NeoGenomics is a cancer reference laboratory that provides cancer testing and partnership programs to pathologists and oncologists. NeoGenomics serves the needs of pathologists, oncologists, academic centers, hospital systems, pharmaceutical firms, integrated service delivery networks, and managed care organizations throughout the United States, and pharmaceutical firms in Europe and Asia.

GH Research PLC is a clinical-stage biopharmaceutical company that aims to transform the treatment of psychiatric and neurological disorders. The company is primarily focused on developing novel and proprietary therapies based on 5-MeO-DMT, a compound known as Mebufotenin, to address the needs of patients suffering from Treatment-Resistant Depression (TRD). Through its innovative approach, GH Research seeks to provide new therapeutic options for individuals who have not responded to conventional treatments.

UroGen Pharma Ltd. is a clinical-stage biopharmaceutical company based in Princeton, New Jersey, focused on developing innovative therapies for specialty cancers and urologic diseases. The company is dedicated to transforming the standard of care in uro-oncology through its diverse pipeline of product candidates. Its lead products, including UGN-101 and UGN-102, aim to ablate tumors non-surgically and address various forms of non-muscle invasive urothelial cancer, such as low-grade upper tract urothelial carcinoma and low-grade non-muscle invasive bladder cancer. UroGen is also advancing UGN-201, an immunotherapy candidate targeting high-grade non-muscle invasive bladder cancer. Furthermore, the company's proprietary RTGel technology enhances drug delivery by enabling sustained release, improving therapeutic efficacy. UroGen has established licensing agreements with Allergan Pharmaceuticals for the development of RTGel-based products and with Agenus Inc. to commercialize treatments for urinary tract cancers. Founded in 2004, UroGen Pharma continues to innovate in the field of urology and oncology.

Visus Therapeutics is a clinical-stage company developing innovative medicines to improve the vision for people around the world.

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.

Biomea Fusion is a privately held precision medicine company with a mission to revolutionize drug development to create more effective therapies for cancer patients. Biomea Fusion has a development portfolio that targets specific gene alterations which occur in the DNA of patients that translate into key drivers of tumor growth. It is Biomea’s goal to move these potentially breakthrough medicines swiftly through the development process and deliver highly impactful treatments for patients in need. The lead program targets the protein-to-protein interaction between menin and the MLL complex for the treatment of various tumors.

Nuance Biotech Inc. is a pharmaceutical development company based in Shanghai, China, founded in 2014. The company specializes in the research and development of various drug delivery methods, including capsules, injections, and tubes. In addition to its focus on drug development, Nuance Biotech offers a commercial and academic platform for pharmaceuticals and provides contract sales organization services to support its initiatives in the healthcare sector.

NiKang Therapeutics is an early-stage biotech company focused on discovering and developing innovative small molecule oncology medicines to help patients with unmet medical needs. Its discovery approach is informed by target structure biology and capitalizes on structure-based drug design enabling a rapid and efficient discovery and development of proprietary drug candidates with the most desirable pharmacological features into clinical studies.

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.

Avadel Pharmaceuticals plc (Nasdaq:AVDL) is a specialty pharmaceutical company that seeks to develop differentiated pharmaceutical products that are safe, effective and easy to take through formulation development, by utilizing its proprietary drug delivery technology and through in-licensing / acquiring new products; ultimately, helping patients adhere to their prescribed medical treatment and see better results. Avadel’s current portfolio of products and product candidates focuses on the urology, central nervous system (CNS) / sleep, and hospital markets. The Company is headquartered in Dublin, Ireland with operations in St. Louis, Missouri and Lyon, France. For more information, please visit www.avadel.com.

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.

Pulmonx Corporation is a medical technology company that specializes in minimally invasive devices for the diagnosis and treatment of chronic obstructive pulmonary disease (COPD), particularly severe emphysema. The company's core offerings include the Zephyr Endobronchial Valve, designed for patients with hyperinflation due to emphysema; the Chartis Pulmonary Assessment System, which assesses collateral ventilation; and the StratX Lung Analysis Platform, a cloud-based service that analyzes computed tomography images to aid in treatment planning. Pulmonx's solutions are intended for patients who remain symptomatic despite medical management and are either not candidates for or do not wish to pursue surgical options. The Zephyr Valve has received FDA pre-market approval and is supported by extensive clinical evidence demonstrating its benefits. Pulmonx serves a global market, with significant operations in the United States and various international regions, and has made a notable impact by treating thousands of patients with its innovative technology.

Orchestra BioMed, Inc. is a biomedical innovation company focused on developing high impact, high value, evidence-based therapeutic solutions for medical procedures that address major medical conditions. Its main focus is on developing new therapies for cardiovascular diseases which are the leading cause of death worldwide, taking the lives of 17.7 million people each year, or 31% of all global deaths according to the World Health Organization. It was founded in 2017 and is headquartered in New Hope, Pennsylvania.

Milestone Pharmaceuticals Inc. is a biopharmaceutical company based in Montréal, Canada, focused on developing and commercializing etripamil for various cardiovascular conditions. Etripamil is a novel short-acting calcium channel blocker designed to be administered as a rapid-onset nasal spray, specifically targeting the termination of episodes of paroxysmal supraventricular tachycardia (PSVT). The company is currently conducting Phase III clinical trials for etripamil in the treatment of PSVT and is also exploring its potential applications for atrial fibrillation, angina, and other related cardiovascular disorders. Founded in 2003, Milestone Pharmaceuticals aims to address unmet medical needs in cardiovascular disease management through innovative therapeutic solutions.

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.

CytomX Therapeutics is an oncology-focused biopharmaceutical company based in South San Francisco, California. The company specializes in developing proteolytically-activated antibody therapeutics using its proprietary Probody technology platform. This innovative approach aims to create cancer immunotherapies that enhance targeting of tumor cells while minimizing effects on healthy tissues. CytomX's clinical-stage product candidates include CX-072, which targets programmed cell death ligand 1; CX-2009, a drug conjugate against CD166; and BMS-986249, a CTLA-4 Probody therapeutic for metastatic melanoma, among others. The company has established strategic collaborations with several major pharmaceutical companies, including AbbVie, Amgen, and Bristol-Myers Squibb, to further the development of its Probody therapeutics. Founded in 2008, CytomX is dedicated to providing patients with more effective and less toxic treatment options for cancer and inflammatory diseases.