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nChroma Bio's integrated product engine allows for safe, accurate, and targeted in vivo administration, addressing major shortcomings of current genetic medicine techniques.

Abeona Therapeutics is a clinical-stage biopharmaceutical company dedicated to developing gene and cell therapies for severe, life-threatening rare genetic diseases. The company's leading programs include EB-101, an autologous, gene-corrected cell therapy for recessive dystrophic epidermolysis bullosa, and ABO-102 and ABO-101, which are AAV-based gene therapies targeting Sanfilippo syndrome types A and B, respectively. Additional therapies under development include ABO-201 and ABO-202 for Batten disease, ABO-401 for cystic fibrosis, and ABO-5OX for genetic eye disorders. Abeona also operates the AIM Vector Platform, which focuses on advancing gene therapy delivery methods using next-generation AAV capsids. The company is engaged in a licensing agreement with REGENXBIO for gene therapies addressing multiple genetic disorders. Founded in 1974 and headquartered in New York, Abeona has a fully operational manufacturing facility that supports its preclinical and clinical research efforts.

Precision BioSciences, Inc. is a biotechnology company based in Durham, North Carolina, focused on genome editing and the development of therapeutic products. Utilizing its proprietary ARCUS genome editing platform, the company aims to create innovative solutions for human diseases and food applications. Precision BioSciences operates through two primary segments: Therapeutic and Food. The Therapeutic segment is engaged in developing allogeneic CAR T immunotherapies, with several candidates in clinical trials targeting various types of cancers, including acute lymphoblastic leukemia and multiple myeloma. Additionally, the company is involved in in vivo gene correction activities and has collaborations for developing treatments for chronic Hepatitis B. The Food segment concentrates on creating nutrition products. Founded in 2006, Precision BioSciences is dedicated to translating advanced gene editing technology into impactful products across the life sciences.

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.

enGene is a developer of a mucosal immunotherapy platform designed to treat inflammatory bowel disease and diabetes. The company's mucosal immunotherapy platform has developed a flexible nucleotide delivery technology targeting mucosal tissues to treat numerous prevalent, chronic diseases via the induction or suppression of protein expression levels, enabling physicians and doctors to regenerate physiologic, meal-regulated insulin secretion from the gut of subjects with diabetes.

PepGen is a clinical-stage biotechnology company based in Oxford, United Kingdom, focused on advancing nucleic acid therapeutics, particularly antisense oligonucleotides. Founded in 2018, the company aims to enhance the delivery and efficacy of these therapies through its proprietary Enhanced Delivery Oligonucleotide (EDO) platform. This innovative platform utilizes engineered peptides designed to improve tissue penetration, cellular uptake, and nuclear delivery of oligonucleotides. PepGen's technology has shown promise in preclinical studies, demonstrating the ability to transport therapeutics effectively into various target tissues, including smooth, skeletal, and cardiac muscle, as well as the central nervous system. The company's mission is to unlock the full potential of nucleic acid therapeutics to transform the treatment landscape for severe neuromuscular and neurological diseases.

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.

Benitec Biopharma is using patented ‘gene-silencing’ technology to develop drugs for chronic and life-threatening human diseases. Their transformational technology, ddRNAi, targets and ‘turns off’ specific genes related to disease, providing lasting treatment and potentially cure with a single dose. Compared to traditional approaches to RNA intereference, ddRNAi is easier to deliver, safer to us, more targeted and more efficient. Benitec has a pipeline of in-house and partnered drug development programs based on ddRNAI, including Hepatitis C and B, cancer-associated pain and drug-resistant lung cancer. Our Hepatitis C program is nearing clinical trials in 2013. ddRNAi has the potential to silence genes related to thousands of human diseases and is protected by over 40 global patents. The technology is available from Benitec under a variety of licence options.

Chroma Medicine is a genomic medicine company based in Cambridge, Massachusetts, founded in 2021. The company focuses on epigenetic editing, aiming to revolutionize the treatment of genetically driven diseases. By utilizing epigenetics, which is nature's inherent mechanism for regulating genes, Chroma Medicine develops programmable epigenetic editors that target specific genes and control chromatin conformation. This innovative approach combines a DNA binding domain with epigenetic effector domains, providing medical practitioners with a new class of therapeutics that offers enhanced control over gene expression.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic 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.

Ceptur Therapeutics is focused on developing innovative genetic medicines aimed at addressing diseases that currently lack effective treatments. The company utilizes its proprietary U1 Adaptor technology, which enables long-lasting and non-genotoxic silencing of disease-causing genes, particularly in challenging tissues beyond the liver and central nervous system. This technology addresses the limitations of existing gene silencing methods and holds promise for cancer treatment, allowing healthcare providers to offer new therapeutic options for patients with various types of cancers.

Chroma Medicine is a genomic medicine company based in Cambridge, Massachusetts, founded in 2021. The company focuses on epigenetic editing, aiming to revolutionize the treatment of genetically driven diseases. By utilizing epigenetics, which is nature's inherent mechanism for regulating genes, Chroma Medicine develops programmable epigenetic editors that target specific genes and control chromatin conformation. This innovative approach combines a DNA binding domain with epigenetic effector domains, providing medical practitioners with a new class of therapeutics that offers enhanced control over gene expression.

LEXEO Therapeutics is a biotechnology company that specializes in the development of gene therapies, particularly those utilizing adeno-associated virus (AAV) technology. The company’s pipeline includes both rare and non-rare monogenic diseases, with a strong emphasis on hereditary and acquired conditions that present significant unmet medical needs. LEXEO's lead programs and preclinical candidates are primarily developed in collaboration with Weill Cornell Medicine’s Department of Genetic Medicine. The company aims to advance its clinical programs toward commercialization while maintaining an ongoing research partnership with Weill Cornell to enhance its preclinical efforts. Through its innovative approach, LEXEO Therapeutics seeks to transform the treatment landscape for diseases that impose a substantial burden on patients and society.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, founded in 2017. The company focuses on developing human genetics-based therapeutics aimed at addressing ineffective red blood cell production in hematologic diseases. Its innovative pipeline includes therapies such as bitopertin for treating erythropoietic porphyrias and Diamond-Blackfan anemia. Disc Medicine targets the hepcidin metabolism axis and aims to modify fundamental biological pathways related to red blood cell formation and function, including heme biosynthesis and iron homeostasis. Through its portfolio of clinical and preclinical product candidates, the company strives to transform the lives of patients suffering from severe hematologic disorders.

Element Biosciences, Inc. is a life science company based in San Diego, California, that specializes in developing innovative genetic analysis tools for research and diagnostics. Established in 2017, the company focuses on creating a modular and high-performing DNA sequencing platform designed to provide high-quality data and workflow flexibility. Their proprietary technology enhances the signal-to-noise ratio and incorporates advancements in surface chemistry, instrumentation, and biochemistry. This approach significantly reduces both operational and capital costs while ensuring superior sequencing data quality, thereby making next-generation sequencing technology more accessible to medical researchers and improving their research capabilities.

TwinStrand Biosciences specializes in advanced DNA sequencing technologies, significantly enhancing the accuracy of mutation detection beyond conventional methods. Their proprietary Duplex Sequencing technology enables the identification of mutations that are often undetectable by standard approaches, making it valuable in various fields such as oncology, infectious disease, organ transplantation, reproductive and genetic health, and forensics. The company is dedicated to improving diagnostic capabilities and advancing research through its innovative sequencing solutions. TwinStrand is also involved in biopharmaceutical development, focusing on the discovery and commercialization of biological drugs aimed at treating life-threatening diseases, particularly cancers. Their product pipeline includes recombinant protein-based prodrugs designed for activation by matrix metalloproteinases associated with solid tumors.

Graphite Bio, Inc. is a clinical-stage gene editing company based in South San Francisco, California, that specializes in targeted DNA integration to develop innovative therapies for severe diseases. The company utilizes a precision gene editing approach to precisely correct genetic mutations, replace malfunctioning genes, and insert therapeutic genetic sequences at specific locations within the genome. Its technology aims to transform treatment paradigms by enabling the targeted integration of genetic payloads, which could potentially cure a range of serious and life-threatening conditions. Graphite Bio was formerly known as Integral Medicines, Inc. before rebranding in August 2020, and it was incorporated in 2019.

DTx Pharma, LLC is a biotechnology company focused on developing and commercializing fatty acid-conjugated peptide therapeutics for patients with rare and chronic diseases. The company specializes in RNA-based therapeutics, utilizing a delivery technology platform that enhances the distribution of nucleic acid drugs to various tissues and organ systems beyond the liver. This innovative approach addresses limitations associated with previous-generation RNA delivery methods, such as poor pharmacokinetics and inadequate cellular uptake. DTx Pharma's products target conditions including retinitis pigmentosa, Duchenne muscular dystrophy, and central nervous system disorders. Founded in 2017 and based in San Diego, California, DTx Pharma aims to provide personalized treatment options across multiple therapeutic areas.

HemoShear Therapeutics, LLC is dedicated to discovering novel biological targets and drug treatments for rare metabolic disorders, particularly focusing on inherited conditions such as Organic Acidemias and Nonalcoholic Steatohepatitis, a chronic liver disease. The company employs its proprietary REVEAL-Tx platform to replicate human disease biology, enabling the development and validation of new therapies. By creating human-relevant systems, HemoShear generates significant human response data that aids pharmaceutical and biotechnology partners in making informed decisions and reducing the risk of costly failures in drug development. Founded in 2008 and based in Charlottesville, Virginia, HemoShear Therapeutics aims to improve the quality of life for patients suffering from metabolic defects that disrupt essential biochemical processes.

Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing innovative therapies for degenerative disorders linked to nucleotide repeat expansions. Founded in 2017 and headquartered in Solana Beach, California, the company is advancing its lead program aimed at treating Friedreich’s ataxia. Additionally, Design Therapeutics is exploring treatments for other conditions such as Fragile X syndrome and myotonic dystrophy, utilizing its proprietary GeneTACTM technology, which involves small-molecule gene-targeted therapeutics designed to modify the underlying causes of these inherited diseases. The company is committed to addressing serious monogenic disorders driven by nucleotide repeat mutations through its ongoing research and development efforts.

Verve Therapeutics, Inc. is a genetic medicines company focused on innovative treatments for cardiovascular disease through gene editing. Founded in 2018 and based in Cambridge, Massachusetts, with a research facility in Philadelphia, the company aims to shift the management of cardiovascular conditions from chronic treatments to single-course gene editing therapies. Its initial programs concentrate on the PCSK9 and ANGPTL3 genes, which are known to significantly impact blood lipid levels, a major factor in cardiovascular disease. Verve Therapeutics collaborates with partners such as Beam Therapeutics and Verily to enhance its gene editing delivery technologies. The company is staffed by a team of experts in cardiovascular medicine, human genetics, and drug development, positioning itself to address the challenges of cardiovascular health effectively.

LEXEO Therapeutics is a biotechnology company that specializes in the development of gene therapies, particularly those utilizing adeno-associated virus (AAV) technology. The company’s pipeline includes both rare and non-rare monogenic diseases, with a strong emphasis on hereditary and acquired conditions that present significant unmet medical needs. LEXEO's lead programs and preclinical candidates are primarily developed in collaboration with Weill Cornell Medicine’s Department of Genetic Medicine. The company aims to advance its clinical programs toward commercialization while maintaining an ongoing research partnership with Weill Cornell to enhance its preclinical efforts. Through its innovative approach, LEXEO Therapeutics seeks to transform the treatment landscape for diseases that impose a substantial burden on patients and society.

Decibel Therapeutics is a developer of hearing disorder therapeutics intended to protect, repair and restore hearing. The company's therapeutics have provided insights into the link between hearing dysfunction and inner ear pathology and it has capabilities to encompass animal models, drug delivery to the inner ear, imaging, inner ear PK/PD modeling and measurement, bioinformatics, genetics and target identification, enabling patients to experience hearing loss therapies with different forms of hearing disorders for their recovery.

Rain Oncology Inc. is a late-stage precision oncology company focused on developing therapies that target oncogenic drivers. The company employs a tumor-agnostic approach, selecting patients based on the underlying genetics of their tumors rather than their histological characteristics. Its leading product candidate, milademetan, is an oral small molecule inhibitor of MDM2, which plays a role in various cancers. In addition to milademetan, Rain Oncology is advancing a preclinical program aimed at inducing synthetic lethality in cancer cells through the inhibition of RAD52. This targeted strategy aims to improve treatment outcomes for patients who are genetically predisposed to benefit from these therapies.

Stoke Therapeutics, Inc. is an early-stage biopharmaceutical company headquartered in Bedford, Massachusetts, focused on developing novel antisense oligonucleotide medicines to address the underlying causes of severe genetic diseases. The company employs its proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) technology to enhance gene expression by targeting RNA splicing, thereby selectively restoring protein levels. Its lead product candidate, STK-001, is currently in clinical testing for the treatment of Dravet syndrome, a serious and progressive genetic epilepsy. Established in 2014, Stoke Therapeutics also collaborates with Invitae Corporation to provide genetic testing services, further supporting its mission to advance gene-targeted therapies for patients with severe genetic conditions.

4D Molecular Therapeutics is a clinical-stage gene therapy company based in Emeryville, California, that specializes in developing innovative gene therapy products to address unmet medical needs. The company utilizes targeted and evolved adeno-associated virus vectors to create a diverse portfolio of product candidates focused on ophthalmology, cardiology, and pulmonology. Among its key projects are 4D-125, currently in a Phase I/II clinical trial for X-linked retinitis pigmentosa; 4D-110, in a Phase I clinical trial for choroideremia; and 4D-310, also in a Phase I/II trial for Fabry disease. Additionally, 4D Molecular Therapeutics is advancing investigational new drug candidates such as 4D-150 for wet age-related macular degeneration and 4D-710 for cystic fibrosis lung disease. Founded in 2013, the company is dedicated to transforming the treatment landscape in these therapeutic areas.