T. Rowe Price Group

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

ArsenalBio is a programmable cell therapy company established in 2019, focused on developing effective and accessible immune cell therapies, primarily targeting cancer treatment. The company utilizes advanced technologies, including CRISPR-based genome engineering, synthetic biology, and machine learning, to create a new paradigm for discovering and developing immune cell therapies. ArsenalBio's innovative approach aims to enhance the efficacy and safety of T-cell therapies while reducing costs for providers and broadening patient access. By employing a full-stack research and development engine, ArsenalBio generates multifunctional T-cell medicines through the precise insertion of large synthetic DNA sequences, enabling clinicians to utilize next-generation autologous T-cell therapies to combat cancer more effectively.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

ADARx Pharmaceuticals, Inc. is a clinical-stage biotechnology company based in San Diego, California, focused on the research and development of RNA editing therapeutics. Founded in 2019, the company specializes in the precise targeting and correction of single point mutations on mRNA transcripts using a proprietary platform that leverages endogenous enzymes known as adenosine deaminase acting on RNA (ADAR). This innovative approach aims to restore the production of functional proteins, addressing genetic mutations that lead to various diseases. ADARx Pharmaceuticals is developing a diverse pipeline of ribonucleic acid targeting therapeutics aimed at treating conditions across multiple therapeutic areas, including genetic disorders, cardiometabolic diseases, complement-mediated diseases, and central nervous system disorders.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

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.

Senti Biosciences is a clinical-stage biotechnology company specializing in synthetic biology and gene circuit engineering for therapeutic purposes. The company employs its Gene Circuits platform to develop innovative therapies that enhance precision in targeting cancer cells while avoiding damage to healthy cells. Senti Bio's approach involves the use of allogeneic chimeric antigen receptor natural killer (CAR-NK) cells that incorporate its gene circuit technologies, aimed at addressing various oncology indications. Among its product candidates are SENTI-202, a Logic Gated off-the-shelf CAR-NK cell therapy designed to selectively eliminate cancer cells without harming healthy bone marrow, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences aims to provide effective treatment options for difficult-to-treat diseases through advanced cell and gene therapies.

Tessera Therapeutics is an early-stage life sciences company pioneering Gene Writing, a new biotechnology designed to offer scientists and clinicians the ability to write small and large therapeutic messages into the genome, thereby curing diseases at their source. Gene Writing holds the potential to become a new category in genetic medicine, building upon recent breakthroughs in gene therapy and gene editing while eliminating important limitations in their reach, utilization, and efficacy. Tessera Therapeutics was founded by Flagship Pioneering, a life sciences innovation enterprise that conceives, resources, and develops first-in-category companies to transform human health and sustainability.

Sionna Therapeutics was formed in late 2019, leveraging 10 years of large pharma scientific investment and expertise. Our experienced scientific team has been on a mission to fully normalize CFTR function. In the three decades since the discovery of the genetic defects that cause cystic fibrosis (CF), researchers have made remarkable advances for patients. Yet the ultimate goal of CF drug development – to fully normalize the function of CFTR, the key protein leading to the pathology of CF – has remained out of reach.

SalioGen Therapeutics is a biotechnology company focused on developing gene therapies for inherited disorders. Utilizing its proprietary Exact DNA Integration Technology (EDIT), the company employs mammal-derived enzymes, known as Saliogase, to facilitate genome engineering. This innovative approach enables the delivery of any size gene through non-viral methods, offering significant advantages in manufacturing and application. SalioGen's technology aims to provide durable, safe, and affordable treatments, potentially transforming the landscape of care for patients with genetic conditions and enhancing the efficiency of cell therapy and biologics manufacturing in the healthcare sector.

DNA Script is a pioneering company in the field of DNA synthesis, focused on enhancing life science and human health through innovative technology. The company has developed a unique enzymatic process that allows for the production of synthetic DNA using natural enzymes. This technology underpins their benchtop DNA printer, SYNTAX, which enables laboratories to synthesize their own oligonucleotides. By doing so, DNA Script provides researchers with greater control over their workflows, facilitating faster access to essential genomic materials for various applications in molecular biology and genomics. This capability accelerates experimentation and supports the development of new therapeutics, positioning DNA Script as a key player in the biotechnology sector.

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.

Arbor Biotechnologies, Inc., founded in 2016 and based in Cambridge, Massachusetts, operates within the biotechnology sector, focusing on bio-discovery and human diagnostic development. The company utilizes a range of advanced technologies, including artificial intelligence, genome sequencing, gene synthesis, and high-throughput screening, to facilitate the discovery of proteins aimed at enhancing human health and sustainability. Arbor's proprietary genomic tools leverage AI and machine learning to analyze genomic sequence data, thereby improving the efficiency of developing curative treatments for genetic diseases. These innovations enable drug developers to replace entire genes and precisely correct mutations, ultimately addressing previously untreatable genetic conditions.

Ginkgo Bioworks is a biotechnology company based in Boston, Massachusetts, founded in 2008. It specializes in biological engineering, designing and developing custom microbes and organisms for various applications. The company discovers and licenses molecules used in flavors, sweeteners, cosmetic ingredients, crop treatments, and pharmaceuticals. Additionally, it offers probiotic bacteria aimed at protecting against infections and creates libraries of molecules for natural product discovery. Ginkgo Bioworks operates primarily in the markets of cultured ingredients, carbon mitigation, probiotics, and biosecurity. The biosecurity segment, which generates the majority of the company's revenue, provides data analytics and related services to organizations across diverse sectors, including food, agriculture, pharmaceuticals, and specialty chemicals.

Ring Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing gene therapies through its innovative Anellovector platform. This platform leverages the biology of the human commensal virome to create redosable and targetable DNA therapies that address several limitations of existing gene therapies, such as restricted access to various tissues, risks associated with genomic integration, and issues with tolerability. Founded in 2017, the company aims to expand the applications of gene therapy beyond traditional gene replacement, targeting a wide range of conditions including genetic disorders, ophthalmology, oncology, and metabolic diseases. By doing so, Ring Therapeutics seeks to provide healthcare solutions that allow for a broader array of therapeutic modalities and improved patient outcomes.

GeneDx is dedicated to providing personalized health insights that enhance diagnosis, treatment, and drug discovery. By integrating extensive genomic expertise with advanced data interpretation capabilities, the company aims to establish precision medicine as a standard in healthcare. GeneDx specializes in exome and genome testing and interpretation, leveraging one of the largest rare disease data sets available. Its operations are divided into two segments: GeneDx, which generates the majority of revenue, and Legacy Sema4 diagnostics. Through these efforts, GeneDx is positioned to significantly impact health outcomes by using genomic and large-scale clinical information.

Prime Medicine is a biotechnology company focused on developing genetic therapies using its proprietary Prime Editing technology. This next-generation gene editing method allows for precise "search and replace" modifications in the genome, aiming to restore normal genetic function and address the underlying causes of various diseases. By targeting a broad spectrum of conditions with significant unmet medical needs, Prime Medicine seeks to revolutionize the field of genetic medicine through effective and efficient therapeutic solutions.

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.

Clear Labs, Inc. is a food analytics company based in Menlo Park, California, founded in 2013. The company specializes in providing a comprehensive analytics platform that analyzes the molecular contents of food and ingredients. Its offerings include Clear Dx™ and Clear Safety™, which utilize next-generation sequencing and data analytics for food safety testing and infectious disease surveillance. Clear Labs serves food manufacturers, suppliers, and retailers by ensuring transparency throughout the supply chain and testing products for various factors such as authenticity, contamination, allergens, and nutritional content. The platform delivers actionable insights that enhance safety and operational efficiency while supporting public health initiatives. Through its integrated genomics system, Clear Labs aims to simplify complex diagnostics across clinical and applied markets, making genomic insights accessible to labs, hospitals, and businesses.

Gyroscope is a clinical-stage gene therapy company that specializes in developing treatments for retinal diseases, particularly focusing on Age-related Macular Degeneration (AMD), a major cause of blindness. The company leverages advancements in understanding the complement system's role in eye diseases and uses gene therapy as a therapeutic approach. Gyroscope's lead investigational therapy, GT005, is designed to be administered as a one-time treatment under the retina, aiming to restore balance to an overactive complement system by enhancing the production of the Complement Factor I protein. Currently, GT005 is undergoing evaluation in multiple clinical trials, including a Phase I/II trial named FOCUS and two Phase II trials called EXPLORE and HORIZON. Founded in 2016 in Stevenage, Herefordshire, by Andrew Lotery, David Kavanagh, and Peter Lachmann, Gyroscope seeks to provide innovative solutions for patients suffering from retinal diseases.

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.

Kronos Bio, Inc. is a clinical-stage biopharmaceutical company dedicated to discovering and developing innovative cancer therapeutics. Founded in 2017 and headquartered in San Mateo, California, the company focuses on targeting dysregulated transcription factors and oncogenic signaling pathways. Its lead candidate, entospletinib, is a selective inhibitor of spleen tyrosine kinase aimed at treating acute myeloid leukemia. Additionally, Kronos Bio is developing KB-0742, an orally bioavailable inhibitor of cyclin-dependent kinase 9, intended for MYC-amplified solid tumors. By leveraging advanced technologies such as high-throughput small-molecule microarrays and targeted protein degradation, Kronos Bio aims to identify effective compounds that can modulate historically undruggable cancer targets, ultimately striving to improve patient outcomes through precision medicine.

Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing innovative gene therapies for both rare and prevalent diseases. Founded in 2016 and originally named Torus Therapeutics, the company focuses on treating conditions primarily affecting the liver and retina, as well as diseases of skeletal muscle, the central nervous system, and oncology. Generation Bio employs a proprietary non-viral platform that includes a high-capacity DNA construct known as closed-ended DNA (ceDNA) and a cell-targeted lipid nanoparticle delivery system (ctLNP). This platform aims to deliver genetic payloads effectively across various tissues and allows for redosable treatments tailored to individual patient needs. Through its advanced manufacturing processes, Generation Bio seeks to expand access to gene therapies and provide durable solutions for patients with both rare and common diseases.

Ginkgo Bioworks is a biotechnology company based in Boston, Massachusetts, founded in 2008. It specializes in biological engineering, designing and developing custom microbes and organisms for various applications. The company discovers and licenses molecules used in flavors, sweeteners, cosmetic ingredients, crop treatments, and pharmaceuticals. Additionally, it offers probiotic bacteria aimed at protecting against infections and creates libraries of molecules for natural product discovery. Ginkgo Bioworks operates primarily in the markets of cultured ingredients, carbon mitigation, probiotics, and biosecurity. The biosecurity segment, which generates the majority of the company's revenue, provides data analytics and related services to organizations across diverse sectors, including food, agriculture, pharmaceuticals, and specialty chemicals.

Allogene Therapeutics is a clinical stage immuno-oncology company. It is mainly engaged in the development and commercialization of genetically engineered allogeneic T cell therapies for the treatment of cancer. The company is involved in developing a pipeline of multiple allogeneic T cell product candidates utilizing validated gene editing and advanced proprietary cell manufacturing technologies. Its pipeline includes UCART19 which is developed for the treatment of relapsed or refractory acute lymphoblastic leukemia (ALL), as well as several preclinical allogeneic CAR T therapies.

Audentes Therapeutics, a clinical-stage biotechnology company based in San Francisco, is dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases resulting from single gene defects. The company is advancing several therapies, including AT132 and AT342, which are in Phase I/II clinical studies for treating X-linked myotubular myopathy and Crigler-Najjar syndrome, respectively. Additionally, it is exploring treatments for Pompe disease and catecholaminergic polymorphic ventricular tachycardia, along with vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Audentes has established collaborative agreements for research and development with Genethon and the University of Pennsylvania. Founded in 2012, Audentes Therapeutics became a subsidiary of Astellas US Holding in 2020 and operates as Astellas Gene Therapies, focusing on leveraging its expertise in gene therapy research and commercialization.

Editas Medicine is a clinical-stage biotechnology company based in Cambridge, Massachusetts, with an additional site in Boulder, Colorado. Founded in 2013, it is focused on discovering and developing innovative genome editing therapeutics to address a wide range of serious diseases. The company utilizes a proprietary platform based on CRISPR/Cas9 technology, which employs a protein-RNA complex to target and modify specific DNA sequences for therapeutic purposes. Editas Medicine aims to translate its advanced genome editing technologies into human therapeutics that can provide precise and corrective molecular modifications to treat genetic disorders. With a strong foundation of patent filings and intellectual property, the company is positioned to advance its early findings into viable treatments for various diseases at the genetic level.

Spark Therapeutics, Inc. is a biotechnology company focused on developing gene therapy products aimed at treating debilitating genetic diseases. Headquartered in Philadelphia, Pennsylvania, the company was founded in 2013 and has established itself as a leader in the field of gene therapy, with a robust pipeline that includes LUXTURNA for retinal dystrophy, as well as several candidates for hemophilia and choroideremia. Spark is also exploring therapies for neurodegenerative diseases and inherited retinal diseases. The company holds a collaboration agreement with Pfizer for the development of hemophilia B treatments and has partnered with Novartis for the commercialization of its lead product outside the United States. Spark Therapeutics leverages a proprietary manufacturing platform and a team with extensive expertise in research, clinical development, and regulatory affairs, building on a legacy of innovation in gene therapy from its origins at The Children’s Hospital of Philadelphia. As of December 2019, Spark operates as a subsidiary of Roche Holding AG.

PacBio designs, develops, and manufactures sequencing systems to resolve genetically complex problems. Its single molecule real-time (SMRT) sequencing technology enables single molecule real-time detection of biological processes.

PacBio designs, develops, and manufactures sequencing systems to resolve genetically complex problems. Its single molecule real-time (SMRT) sequencing technology enables single molecule real-time detection of biological processes.