RTW Investments

Tenax Therapeutics, Inc. is a specialty pharmaceutical company dedicated to the identification, development, and commercialization of products aimed at addressing cardiovascular and pulmonary diseases with significant unmet medical needs. Headquartered in Morrisville, North Carolina, Tenax focuses on levosimendan, a pharmaceutical product designed to reduce morbidity and mortality in cardiac surgery patients at risk for low cardiac output syndrome. The company is also actively conducting a Phase 2 clinical trial to explore the efficacy of levosimendan in treating pulmonary hypertension associated with heart failure with preserved ejection fraction. Additionally, Tenax is involved in developing novel formulations of imatinib mesylate, a kinase inhibitor with the potential to serve as a disease-modifying therapy for pulmonary arterial hypertension. Founded in 1967, Tenax Therapeutics underscores its commitment to advancing treatments within these critical therapeutic areas.

Windward Bio is a clinical-stage drug development company focused on improving outcomes for individuals with advanced immunological diseases, particularly severe respiratory conditions. The company is advancing a monoclonal antibody targeting thymic stromal lymphopoietin (TSLP) into phase 2 development while creating innovative bispecific programs for various immunological disorders. Led by a team of biopharmaceutical executives, Windward Bio aims to address significant unmet medical needs in this field.

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.

Ottimo Pharma focuses on creating pioneering cancer treatments for solid tumors, specifically through its lead program, Jankistomig, a bi-functional antibody targeting immune checkpoints and angiogenesis. The company aims to enhance treatment outcomes and alleviate healthcare burdens by advancing this dual-pathway approach.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

Neurogene is a developer of generic medicines intended to improve patient outcomes for neurological disorders.The company's offerings include advancing genetic medicines for many neurological disorders where no effective treatment options exist, providing patients and families affected by rare neurological disorders with gene therapy that improves quality of life.

Evommune, Inc. is a biotechnology company based in Los Altos, California, that focuses on developing and manufacturing tissue-based medicines aimed at treating inflammatory diseases. Founded in 2020, the company employs a proprietary tissue-based platform to accelerate the discovery of novel therapies, with a particular emphasis on immunology and dermatology. Evommune's approach seeks not only to alleviate the symptoms of inflammatory disorders but also to halt disease progression, thereby improving the quality of life for patients. Its pipeline includes initiatives targeting prevalent inflammatory conditions, with a commitment to advancing patient care by creating best-in-class treatment options. Through innovative strategies, Evommune aims to drive meaningful advancements in the field of inflammatory disease management.

Kailera Therapeutics is a clinical-stage biotechnology company that provides revolutionary therapies for treating obesity and related diseases. Kailera Therapeutics is advancing a range of injectable and oral GLP-1 (Glucagon-like peptide-1) therapies for obesity and related diseases.

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.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

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.

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

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.

Abdera Therapeutics is an oncology company focused on developing targeted alpha therapies (TATs) for patients suffering from relapsed, refractory, and metastatic cancers. The company specializes in targeted radiotherapies that employ purpose-built vectors to deliver high-energy radioisotopes directly to tumors and metastatic lesions. This innovative approach aims to provide more effective treatment options for patients, capitalizing on the significant therapeutic and commercial potential of TATs. With the nuclear medicine market projected to grow substantially, Abdera's targeted therapies are positioned to play a crucial role in the evolving landscape of cancer treatment.

Cargo Therapeutics is a clinical-stage biotechnology company focused on developing next-generation CAR T-cell therapies aimed at overcoming cancer treatment resistance and improving patient access to potentially curative options. The company aims to enhance treatment outcomes by addressing the limitations of existing therapies, which currently provide curative benefits to less than half of cancer patients. Its lead program, CRG-022, is a novel CAR T-cell product candidate that targets CD22, a tumor antigen prevalent in many B-cell malignancies. By tackling issues such as limited durability, safety concerns, and supply chain obstacles, Cargo Therapeutics is committed to advancing innovative solutions that make effective cancer treatments more accessible to patients.

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.

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.

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.

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

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

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.

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.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

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

Inbrace is a medical device company focused on innovative orthodontic solutions. Its flagship product, INBRACE, is a discreet alignment system that is placed behind the teeth, utilizing patented self-guiding technology to apply continuous forces for gentle correction of dental alignment. This approach significantly reduces pain and minimizes the number of required visits to the orthodontist, addressing common patient concerns and enhancing satisfaction. INBRACE is FDA registered and aims to expand access to orthodontic treatment, thereby driving practice growth for dental professionals. The company's platform incorporates digital treatment planning, computer modeling, and direct digital manufacturing, allowing for customized solutions that maintain high standards of care across various orthodontic cases.

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.

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.

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.

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

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.

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.

Prometheus Biosciences, formerly known as Prometheus Laboratories, is a specialty pharmaceutical company headquartered in San Diego, California. The company develops and commercializes innovative pharmaceutical and diagnostic products aimed at enhancing individualized patient care, particularly in the fields of gastroenterology and autoimmune diseases. Prometheus offers a range of diagnostic tests, including assays for monitoring drug and anti-drug antibody levels, as well as tests for inflammatory bowel disease and celiac disease. Among its marketed products are Entocort EC, indicated for Crohn's disease, and Imuran, used in kidney transplantation and rheumatoid arthritis management. Additionally, the company is involved in developing new treatments, such as COLAL-PRED, for gastrointestinal disorders. Through its advanced diagnostics and therapeutic offerings, Prometheus Biosciences assists healthcare providers in optimizing treatment strategies and improving patient outcomes.

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.

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.

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.

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.

Landos Biopharma is a developer of an oral first-in-class therapeutics designed to target autoimmune diseases.The company's therapeutics are novel, locally-acting small molecules targeting inflammatory bowel disease (IBD), crohn's disease (CD) and ulcerative colitis (UC), enabling healthcare providers to cure their patients.

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

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.

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.

Beta Bionics is a biotechnology company focused on developing an integrated bionic pancreas system known as the iLet. This innovative device aims to alleviate the challenges and costs associated with diabetes care by providing a dual-chamber infusion pump that delivers insulin and glucagon with precision. The iLet autonomously calculates and administers the necessary doses, enabling patients to effectively manage their blood sugar levels. The system has received breakthrough designation for all its configurations, including insulin-only, glucagon-only, and bihormonal options, and is compatible with Zealand Pharma’s dasiglucagon, a glucagon analogue known for its stability in a ready-to-use aqueous solution. Through this technology, Beta Bionics seeks to improve the quality of life for individuals living with diabetes.

Roivant Sciences is a biopharmaceutical company dedicated to advancing the development of innovative late-stage drug candidates and enhancing healthcare delivery. The company aims to expedite the process of bringing new medicines to patients while reducing associated costs, thereby benefiting the healthcare system. Roivant achieves this by creating specialized entities known as "Vants," which are agile biotech and healthcare technology companies that leverage unique talent sourcing and incentive alignment strategies. In addition to its focus on biopharmaceuticals, Roivant incubates discovery-stage companies and health technology startups that complement its core business. Among its commercial-stage drug candidates is VTAMA, a treatment for plaque psoriasis in adults, along with several other candidates, including Batoclimab, IMVT-1402, Brepocitinib, and Namilumab, which are at various stages of development.

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.

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.