RTW Investments

Neurogene is a clinical-stage biotechnology company focused on developing genetic medicines for rare neurological diseases. It leverages its proprietary transgene regulation platform, EXACT, to create gene therapies with the aim of addressing high-need conditions. The company’s pipeline includes NGN-401 for Rett syndrome and NGN-101 for CLN5 Batten disease. Founded in 2018, Neurogene is headquartered in New York and concentrates on advancing therapies that can improve outcomes for patients with limited treatment options.

Santa Ana Bio is a biotechnology company specializing in the development of precision immunology therapies for autoimmune diseases. It employs advanced multi-omics platforms and antibody engineering techniques to design targeted biologics, aiming to improve patient outcomes and quality of life.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

BioAge Labs develops medicines to treat aging and age-related diseases using its proprietary BioAge platform, which combines systems biology and AI. Its pipeline includes BGE-117 for multiple aging-related conditions, BGE-175 for immune aging and respiratory infections, and azelaprag for obesity in the elderly.

Taysha Gene Therapies develops adeno-associated virus-based gene therapies to treat monogenic diseases of the central nervous system. Its pipeline includes treatments for GM2 gangliosidosis, CLN1 disease, Rett syndrome, SLC6A1 haploinsufficiency disorder, and Surfeit locus 1 deficiency. The company collaborates with The University of Texas Southwestern Medical Center to develop and commercialize transformative gene therapy treatments.

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 is a biotechnology company focused on developing gene therapies for rare pediatric diseases. Its clinical-stage programs include treatments for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Infantile Malignant Osteopetrosis.

Monte Rosa Therapeutics is a biotechnology company focused on cancer therapeutics that target protein degradation pathways. It specializes in developing molecular glue degraders, a class of small molecules that direct disease-relevant proteins to degradation by the cell's natural machinery. The company uses its QuEEN platform, incorporating artificial intelligence and proprietary experimental tools, to identify target proteins for degradation by molecular glue degraders. It maintains a diverse library of more than 50,000 molecules and advancing its pipeline, including MRT-2359, a candidate that targets the translation termination factor GSPT1 for potential use in MYC-driven tumors.

Biomea Fusion is a precision medicine company focused on accelerating cancer drug development by targeting specific gene alterations that drive tumor growth. Its lead program aims to disrupt the protein-to-protein interaction between menin and the MLL complex for treating various tumors.

Encoded Therapeutics is a biotechnology company focused on precision gene therapies to treat a broad range of severe genetic disorders. It develops therapies and a platform that identifies human genome sequences that regulate gene expression using screening and computational approaches. The company's pipeline targets conditions across multiple pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular disease, aiming to help medical practitioners treat patients and improve outcomes. Based in South San Francisco, California, Encoded Therapeutics was founded in 2014 and was previously known as Encoded Genomics.

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 is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

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 is a biotechnology company focused on precision gene therapies to treat a broad range of severe genetic disorders. It develops therapies and a platform that identifies human genome sequences that regulate gene expression using screening and computational approaches. The company's pipeline targets conditions across multiple pathways, including neurocircuitry, liver and metabolic disorders, neurodegeneration, and cardiovascular disease, aiming to help medical practitioners treat patients and improve outcomes. Based in South San Francisco, California, Encoded Therapeutics was founded in 2014 and was previously known as Encoded Genomics.

Stoke Therapeutics is a biopharmaceutical company developing novel antisense oligonucleotide medicines. These target RNA splicing to increase gene expression, aiming to treat severe genetic diseases. Its lead product candidate, STK-001, focuses on Dravet syndrome, a progressive genetic epilepsy.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

Orchard Therapeutics is a UK-based biopharmaceutical company dedicated to developing and commercializing innovative gene therapies for rare diseases. Its core focus is autologous ex vivo gene therapy, transforming hematopoietic stem cells into a gene-modified drug product for single-administration treatments. The company's portfolio includes Strimvelis, approved by the EMA in 2016, and several programs in advanced registrational studies across various disease areas.

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.