RTW Investments

Neurogene Inc., established in 2018 and headquartered in New York, specializes in developing genetic medicines for treating neurological disorders. The company focuses on utilizing adeno-associated virus (AAV) vectors to deliver therapeutic genes for conditions such as Charcot-Marie Tooth disease type 4J (CMT4J), Aspartylglucosaminuria (AGU), and an undisclosed Lysosomal Storage Disease (LSD). Neurogene aims to provide effective treatment options where none currently exist, aiming to improve the quality of life for patients and families affected by these rare neurological disorders.

Santa Ana Bio is a biotechnology company focused on developing precision biologics for autoimmune and inflammatory diseases. The company employs advanced technologies, including deep cell profiling, multi-omics platforms, and single-cell genomics, to create targeted therapies. By utilizing antibody engineering and sophisticated analytics, Santa Ana Bio aims to deliver personalized treatments that enhance patient outcomes and improve quality of life for those affected by autoimmune conditions.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

BioAge Labs, Inc. is a biotechnology company focused on developing therapies to treat aging and age-related diseases. The company utilizes a proprietary systems biology and artificial intelligence platform to identify significant drug targets that influence aging, leveraging extensive datasets to uncover the molecular drivers of age-related pathologies. Among its key offerings is BGE-117, a hypoxia-inducible factor prolyl hydroxylase inhibitor that activates HIF-1 target genes involved in various biological processes, including tissue regeneration and vascular remodeling. Another notable product is BGE-175, an orally administered inhibitor targeting the prostaglandin D2 DP1 signaling pathway, which is linked to an increased risk of mortality and infections, including those associated with immune aging. Founded in 2015 and headquartered in Richmond, California, BioAge Labs is dedicated to harnessing the biology of human aging to create innovative therapies for metabolic and age-related diseases.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, focused on developing adeno-associated virus (AAV)-based therapies for monogenic diseases of the central nervous system (CNS). Founded in 2019, the company aims to create curative treatments for severe and life-threatening conditions. Its pipeline includes several product candidates, 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 collaborates with The University of Texas Southwestern Medical Center to enhance its development and commercialization efforts, leveraging expertise in gene therapy to advance its mission of eradicating monogenic CNS 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, the company utilizes advanced T cell engineering and synthetic biology technologies to target and eliminate autoreactive immune cells that contribute to inflammatory conditions. By addressing the underlying causes of these diseases, Kyverna Therapeutics aims to offer effective solutions for patients suffering from autoimmune disorders.

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, the company utilizes advanced T cell engineering and synthetic biology technologies to target and eliminate autoreactive immune cells that contribute to inflammatory conditions. By addressing the underlying causes of these diseases, Kyverna Therapeutics aims to offer effective solutions for patients suffering from autoimmune disorders.

Rocket Pharmaceuticals, Inc. is a biotechnology company dedicated to developing innovative gene therapies for rare and severe pediatric diseases. The company specializes in lentiviral vector and adeno-associated virus platforms to target genetic disorders. Its lead program focuses on treating Fanconi Anemia, a serious condition that leads to bone marrow failure. In addition to this, Rocket is actively researching therapies for several other bone marrow-derived disorders, including Pyruvate Kinase Deficiency, Leukocyte Adhesion Deficiency-I, and Infantile Malignant Osteopetrosis. Furthermore, the company is developing an AAV-based gene therapy for Danon Disease, a multi-organ disorder associated with increased risk of cardiovascular complications. To advance its research, Rocket Pharmaceuticals maintains numerous collaborations and license agreements with prestigious institutions, enhancing its capabilities in gene therapy development. The company is headquartered in New York, New York.

Monte Rosa Therapeutics is a biotechnology company dedicated to developing innovative cancer therapeutics through the modulation of protein degradation pathways. Specializing in molecular glue degraders (MGDs), the company utilizes the body's natural mechanisms for protein destruction to selectively degrade proteins that are relevant to therapeutic outcomes. Employing its QuEEN platform, which integrates artificial intelligence and proprietary experimental tools, Monte Rosa identifies target proteins for degradation by MGDs from its extensive library of over 50,000 molecules. The company's most advanced product candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for the treatment of MYC-driven tumors. Monte Rosa Therapeutics aims to deliver pioneering therapies for cancer and potentially other diseases through its innovative approaches.

Biomea Fusion is a privately held biopharmaceutical company focused on developing precision medicines for cancer patients. The company targets specific gene alterations that drive tumor growth, aiming to create more effective therapies. Its lead program focuses on inhibiting the interaction between menin and the MLL complex, which is crucial in various tumors. Biomea Fusion's goal is to expedite the development process of these potentially groundbreaking medicines to deliver impactful treatments for patients with genetically defined cancers.

Encoded Therapeutics, Inc. is a biotechnology company focused on developing precision gene therapies for severe genetic disorders. Utilizing its proprietary platform, the company identifies genomic sequences that regulate gene expression, facilitating advancements in viral gene therapy. Encoded Therapeutics has a diverse therapy pipeline targeting various genetic and acquired disorders across multiple disease pathways, including neurocircuitry, metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and headquartered in South San Francisco, California, the company is dedicated to improving patient outcomes through innovative gene therapy solutions.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Cogent Biosciences is a biotechnology company dedicated to developing precision therapies for genetically defined diseases, aiming to treat the underlying causes and improve patient lives. The company employs proprietary T-cell engineering technology alongside tumor-targeting antibodies to harness the body's immune system against cancer. One of its key programs, CGT9486, is a selective tyrosine kinase inhibitor designed to inhibit specific mutations in KIT exon 17, which are associated with Systemic Mastocytosis and advanced gastrointestinal stromal tumors (GIST).

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Frequency Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to repairing or reversing damage caused by degenerative diseases through its innovative Progenitor Cell Activation approach. This method utilizes a combination of small molecules to activate progenitor cells in the body, which facilitates the creation of functional tissue without the complexities of genetic engineering. The company’s lead product candidate, FX-322, is currently undergoing phase 2a clinical trials aimed at treating the underlying causes of sensorineural hearing loss. In addition to FX-322, Frequency Therapeutics is exploring treatments for various degenerative conditions, including multiple sclerosis and diseases affecting muscle, the gastrointestinal tract, skin, and bone. Established in 2014 and based in Woburn, Massachusetts, Frequency Therapeutics has established a collaboration agreement with Astellas Pharma Inc. for the development and commercialization of FX-322.

Encoded Therapeutics, Inc. is a biotechnology company focused on developing precision gene therapies for severe genetic disorders. Utilizing its proprietary platform, the company identifies genomic sequences that regulate gene expression, facilitating advancements in viral gene therapy. Encoded Therapeutics has a diverse therapy pipeline targeting various genetic and acquired disorders across multiple disease pathways, including neurocircuitry, metabolic disorders, neurodegeneration, and cardiovascular diseases. Founded in 2014 and headquartered in South San Francisco, California, the company is dedicated to improving patient outcomes through innovative gene therapy solutions.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company focused on developing novel antisense oligonucleotide medicines aimed at addressing the underlying causes of severe genetic diseases. Founded in 2014 and headquartered in Bedford, Massachusetts, the company utilizes its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach to enhance gene expression through RNA splicing. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Stoke Therapeutics has also partnered with Invitae Corporation to offer complementary genetic testing services.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Orchard Therapeutics is a biopharmaceutical company based in London, focused on developing innovative gene therapies for serious and life-threatening rare diseases. The company employs an autologous ex vivo gene therapy approach, which aims to modify a patient's hematopoietic stem cells to create a personalized treatment through a single administration. Orchard's product portfolio includes Strimvelis, the first gene therapy approved by the European Medicines Agency for adenosine deaminase-severe combined immunodeficiency (ADA-SCID). The company is advancing several clinical programs, including OTL-101 for ADA-SCID, OTL-200 for metachromatic leukodystrophy, OTL-103 for Wiskott-Aldrich syndrome, OTL-102 for X-linked chronic granulomatous disease, and OTL-300 for transfusion-dependent beta-thalassemia. Additionally, Orchard has a robust preclinical pipeline targeting various mucopolysaccharidosis types. Founded in 2015, Orchard Therapeutics collaborates with leading institutions in the field to enhance its research and development efforts.

Solid Biosciences Inc. is a life science company focused on developing therapies for Duchenne muscular dystrophy (DMD), a 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. In addition to SGT-001, Solid Biosciences is advancing the Anti-LTBP4 program, which involves a monoclonal antibody designed to reduce fibrosis and inflammation by targeting the LTBP4 protein. The company also develops biomarkers and sensors, as well as the Solid Suit program, which focuses on creating wearable assistive devices with functional and therapeutic benefits. Founded in 2013 and headquartered in Cambridge, Massachusetts, Solid Biosciences has established a strategic collaboration with Ultragenyx to enhance gene therapy development for DMD. With no current cure for DMD, the company aims to provide innovative solutions for a condition that affects an estimated 10,000 to 15,000 cases in the United States.

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