Senti Biosciences is a clinical-stage biotechnology company based in South San Francisco, California, focused on developing innovative gene and cell therapies using its proprietary synthetic biology platform. Founded in 2016, the company aims to address complex diseases through advanced therapeutic solutions. Senti's technology includes engineered allogeneic chimeric antigen receptor natural killer (CAR-NK) cells, which are designed to target and eliminate cancer cells while minimizing damage to healthy tissue. Among its product candidates are SENTI-202, a Logic Gated CAR-NK cell therapy intended for cancer treatment, and SENTI-301A, which targets hepatocellular carcinoma. Senti Biosciences operates as a resident company at Johnson & Johnson Innovation's JLABS in South San Francisco, collaborating with experts in synthetic biology and computation to advance its therapeutic developments.
Tessera Therapeutics is an early-stage life sciences company focused on pioneering Gene Writing technology, which allows for the permanent modification of the human genome. This innovative approach enables scientists and clinicians to introduce therapeutic messages into the genome, offering a potential cure for diseases at their source. By building on recent advancements in gene therapy and gene editing, Tessera Therapeutics aims to address significant limitations in these areas, enhancing their applicability and effectiveness. Founded by Flagship Pioneering, the company is positioned to transform genetic medicine and improve patient care through its groundbreaking technology.
Developer of life sciences technology intended to develop highly effective and differentiated treatments for cystic fibrosis. The company is developing first-in-class small molecules that are focused on a unique target with the potential to fully normalize CFTR function for the first time for the vast majority of people living with cystic fibrosis, enabling patients with best-in-class efficacy and reach and long-term benefit for people with cystic fibrosis.
SalioGen Therapeutics advances in curative genetic therapies using its Exact DNA Integration Technology (EDIT) platform, a mammal-derived genome engineering technology. It is focused on providing durable, safe, and affordable non-viral gene therapies to more patients with inherited diseases. Looking ahead, SalioGen will also explore the EDIT platform’s capabilities to streamline cell therapy, cell engineering, and biologics manufacturing to help an even broader population of patients in need.
DNA Script SAS, founded in 2014 and based in Paris, France, specializes in synthetic biology tools and innovative DNA synthesis technology. The company has developed a groundbreaking enzymatic technology that powers the SYNTAX, the world's first benchtop DNA printer. This device allows laboratories to produce synthetic oligonucleotides on-site, significantly enhancing workflow control and expediting access to results. By employing an efficient biochemical process for nucleic acid synthesis, DNA Script enables researchers to rapidly generate essential genomic materials, such as oligonucleotides, which are crucial for genomics and molecular biology applications. Through its advancements, DNA Script aims to facilitate breakthroughs in life sciences and human health.
Generate Biomedicines, Inc. specializes in developing a generative biology platform aimed at inventing novel therapeutic proteins, including antibodies, peptides, enzymes, and other related compounds. Utilizing advanced machine learning techniques, the platform analyzes the genetic code that determines protein function, allowing for the creation of new protein sequences optimized for therapeutic applications. This innovative approach enables the company to generate proteins that interact specifically and effectively with targeted therapeutic needs, facilitating the development of new drugs across various protein modalities. Founded in 2018 and located in Cambridge, Massachusetts, Generate Biomedicines was previously known as Generate Biologics, Inc. until its name change in March 2020.
Chroma Medicine is a genomic medicine company focused on epigenetic editing to transform the treatment of genetically driven diseases. By leveraging epigenetics, which regulates gene expression naturally, Chroma Medicine aims to develop innovative therapies that provide precise control over gene activity. The company utilizes programmable epigenetic editors that combine DNA binding domains with epigenetic effector domains, allowing for targeted manipulation of genes and chromatin structure. This approach promises to create a new class of therapeutics that offers unparalleled control over gene expression, potentially leading to more effective treatments for a range of genetic disorders.
Ginkgo Bioworks, founded in 2008 and based in Boston, Massachusetts, specializes in biological engineering by designing, developing, and licensing custom microbes and organisms. The company focuses on discovering and producing a variety of molecules for applications in flavors, sweeteners, cosmetic ingredients, crop treatments, and pharmaceuticals. Additionally, Ginkgo Bioworks offers probiotic bacteria aimed at protecting against harmful infections and creates libraries of molecules for various uses. Its market reach includes sectors such as cultured ingredients, carbon mitigation, probiotics, and natural product discovery.
Ring Therapeutics, Inc. is a biotechnology company that specializes in developing gene therapies through its innovative viral vector platform, Anellovector. This platform leverages the human commensal virome to create redosable and targetable DNA therapies, addressing key limitations of existing gene and DNA treatments, such as restricted access to diverse tissues and challenges with tolerability and genomic integration. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics aims to expand the scope of gene therapy beyond traditional gene replacement, enabling a broader range of applications across various medical fields, including genetic disorders, ophthalmology, oncology, and metabolic diseases. By focusing on cellular and tissue specificity, the company aspires to unlock new therapeutic possibilities for previously inaccessible conditions.
Prime Medicine Inc is a biotechnology company committed to delivering genetic therapies to address diseases by deploying gene editing technology, Prime Editing. The Prime Editing technology is a next-generation technology that can search and replace to restore normal genetic function in the genome and can treat a wide spectrum of diseases with high unmet medical needs and efficient and broad gene editing technology.
Element Biosciences, Inc. develops genetic analysis tools for the research and diagnostic markets. The company provides a disruptive DNA sequencing technology and a sequencing platform, such as surface chemistry, sequencing chemistry, detection, and data analysis. The company was incorporated in 2017 and is based in San Diego, California.
Scribe Therapeutics Inc. is a biotechnology company focused on developing innovative therapeutics using CRISPR technology to address genetic disorders. Founded in 2017 and based in Berkeley, California, Scribe specializes in engineering CRISPR enzymes known as X-Editing (XE) molecules, which enhance the efficacy, specificity, and deliverability of genome editing compared to existing methods. The company's proprietary platform enables the creation of custom-engineered enzymes and delivery systems, facilitating precision targeting within the genome. Scribe Therapeutics aims to establish CRISPR-based therapies as a standard in clinical care, expanding access to transformative treatments for a wide range of conditions, including immuno-oncology and degenerative disorders. Through its commitment to overcoming the limitations of current genome editing technologies, Scribe Therapeutics is poised to make substantial contributions to human therapeutics.
Design Therapeutics, Inc. is a clinical-stage biotechnology company based in Solana Beach, California, focused on developing innovative therapies for degenerative disorders resulting from nucleotide repeat expansions. Founded in 2017, the company is advancing its lead program aimed at treating Friedreich's ataxia, while also exploring treatment options for other conditions such as Fragile X syndrome and myotonic dystrophy. Design Therapeutics is known for its GeneTACTM molecules, a new class of small-molecule gene-targeted therapies designed to address the underlying causes of diseases linked to inherited nucleotide repeat expansion mutations. The company's ongoing research efforts seek to develop disease-modifying treatments for serious conditions driven by these genetic factors.
Color is a population health technology company founded in 2014 and based in Burlingame, California. It focuses on providing technology and infrastructure for large-scale health initiatives, making advanced healthcare services accessible and cost-effective. Color's Population Genomics Platform offers modular and comprehensive solutions designed to help health systems, employers, and research institutions develop and implement effective population health programs. The company combines innovative technology with specialized laboratory infrastructure and clinical services, facilitating various aspects of health programs, including strategy, participant engagement, genetic testing, data management, and clinical reporting. Color’s partnerships with organizations such as the National Institutes of Health and Verily enhance its capabilities, enabling it to deliver precision health programs that address the challenges of traditional healthcare access and costs.
Color is a population health technology company founded in 2014 and based in Burlingame, California. It focuses on providing technology and infrastructure for large-scale health initiatives, making advanced healthcare services accessible and cost-effective. Color's Population Genomics Platform offers modular and comprehensive solutions designed to help health systems, employers, and research institutions develop and implement effective population health programs. The company combines innovative technology with specialized laboratory infrastructure and clinical services, facilitating various aspects of health programs, including strategy, participant engagement, genetic testing, data management, and clinical reporting. Color’s partnerships with organizations such as the National Institutes of Health and Verily enhance its capabilities, enabling it to deliver precision health programs that address the challenges of traditional healthcare access and costs.
Generation Bio is a genetic medicines company based in Cambridge, Massachusetts, that specializes in developing gene therapies aimed at treating both rare and prevalent diseases. Established in 2016, the company focuses on conditions affecting the liver, retina, skeletal muscle, central nervous system, and oncology. Generation Bio employs an innovative non-viral platform that includes a proprietary high-capacity DNA construct known as closed-ended DNA (ceDNA), a cell-targeted lipid nanoparticle delivery system (ctLNP), and a scalable, capsid-free manufacturing process. This approach allows for the targeted delivery of genetic materials, including large and multiple genes across various tissues, and enables redosable treatments tailored to individual patient needs. By enhancing access to therapies for rare diseases and addressing more common conditions, Generation Bio aims to provide durable and sustainable treatment options for patients throughout their lives.
Ginkgo Bioworks, founded in 2008 and based in Boston, Massachusetts, specializes in biological engineering by designing, developing, and licensing custom microbes and organisms. The company focuses on discovering and producing a variety of molecules for applications in flavors, sweeteners, cosmetic ingredients, crop treatments, and pharmaceuticals. Additionally, Ginkgo Bioworks offers probiotic bacteria aimed at protecting against harmful infections and creates libraries of molecules for various uses. Its market reach includes sectors such as cultured ingredients, carbon mitigation, probiotics, and natural product discovery.
Ginkgo Bioworks, founded in 2008 and based in Boston, Massachusetts, specializes in biological engineering by designing, developing, and licensing custom microbes and organisms. The company focuses on discovering and producing a variety of molecules for applications in flavors, sweeteners, cosmetic ingredients, crop treatments, and pharmaceuticals. Additionally, Ginkgo Bioworks offers probiotic bacteria aimed at protecting against harmful infections and creates libraries of molecules for various uses. Its market reach includes sectors such as cultured ingredients, carbon mitigation, probiotics, and natural product discovery.
Allogene Therapeutics is a clinical-stage biotechnology company focused on immuno-oncology, particularly the development of genetically engineered allogeneic T cell therapies for cancer treatment. The company is advancing a pipeline of allogeneic T cell product candidates that leverage validated gene editing and proprietary cell manufacturing technologies. Key offerings include UCART19, which targets relapsed or refractory acute lymphoblastic leukemia, alongside several preclinical allogeneic CAR T therapies aimed at various malignancies and autoimmune diseases. By using T cells derived from healthy donors, Allogene Therapeutics aims to provide off-the-shelf treatment options that enhance patient eligibility and scalability. The company seeks to redefine CAR T therapy through its innovative approaches, addressing conditions like lymphoma, leukemia, and solid tumors.
Audentes Therapeutics is a clinical-stage biotechnology company dedicated to developing and commercializing gene therapy products for patients with serious, life-threatening rare diseases caused by single gene defects. The company is advancing several therapies, including AT132, currently in Phase I/II clinical trials for X-linked myotubular myopathy, and AT342, also in Phase I/II studies for Crigler-Najjar syndrome. Additionally, Audentes is exploring AT845 in preclinical studies for Pompe disease and AT307 for the CASQ2 subtype of catecholaminergic polymorphic ventricular tachycardia. The company is also working on vectorized antisense treatments for Duchenne muscular dystrophy and myotonic dystrophy type 1. Collaborations include a partnership with Genethon for XLMTM treatments and an agreement with the University of Pennsylvania for Crigler-Najjar syndrome products. Founded in 2012 and headquartered in San Francisco, California, Audentes Therapeutics operates as a subsidiary of Astellas US Holding, Inc.
Based in Dallas, Texas, AveXis is a clinic-ready, synthetic biology platform company. AveXis has, at its core, a desire to establish unique industry and research alliances which will bring innovative treatments to people with unmet medical needs. Our work in spinal muscular atrophy (SMA), a rare/orphan disease, is our first focus.
Editas Medicine is a clinical-stage company focused on developing innovative genome editing therapeutics to address serious diseases at the genetic level. Established in 2013 and headquartered in Cambridge, Massachusetts, with an additional site in Boulder, Colorado, the company leverages its proprietary CRISPR/Cas9 technology to create precise and corrective molecular modifications. This approach utilizes a protein-RNA complex, which includes the Cas9 enzyme and a guide RNA, to target and repair specific DNA sequences. Editas Medicine aims to translate its advances in genome editing into transformative human therapies, supported by a robust portfolio of patents and intellectual property that underpins its foundational technologies.
BeiGene, Ltd. is a biotechnology company engaged in the development and commercialization of targeted and immuno-oncology therapies for cancer. Founded in 2010 and based in Beijing, the company operates globally, focusing on addressing significant unmet medical needs across various cancer types. Its commercial portfolio includes treatments such as BRUKINSA for mantle cell lymphoma and Tislelizumab for Hodgkin's lymphoma, alongside other therapies for conditions like breast cancer and multiple myeloma. BeiGene's pipeline features several clinical-stage drug candidates, including Zanubrutinib, an inhibitor targeting Bruton's tyrosine kinase, and Pamiparib, a PARP inhibitor. The company is dedicated to making innovative cancer treatments more affordable and accessible, and it collaborates with various pharmaceutical firms to enhance its research and development efforts.
Spark Therapeutics, Inc. is dedicated to developing gene therapy products aimed at treating debilitating genetic diseases. The company is known for LUXTURNA, a treatment for patients with biallelic RPE65 mutation-associated retinal dystrophy, and has an extensive pipeline that includes candidates like SPK-8011 and SPK-8016 for hemophilia, SPK-7001 for choroideremia, and SPK-9001 for hemophilia B. Additionally, Spark is exploring liver-directed gene therapies such as SPK-3006 for Pompe disease and has programs targeting neurodegenerative diseases, including Huntington's disease and a form of Batten disease. Spark Therapeutics also engages in collaborations, notably with Pfizer for the SPK-FIX program for hemophilia B, and has a licensing agreement with Novartis for the commercialization of LUXTURNA outside the United States. Established in 2013 and based in Philadelphia, Spark Therapeutics operates as a subsidiary of Roche Holding AG and leverages a proprietary manufacturing platform to support its clinical studies across various therapeutic areas.
Pacific Biosciences designs, develops, and manufactures advanced sequencing systems that address complex genetic challenges. Utilizing single molecule real-time (SMRT) sequencing technology, it enables the real-time detection of biological processes at the single-molecule level. The company offers products such as the PacBio RS II and Sequel Systems, which are capable of conducting, monitoring, and analyzing biochemical reactions in real time. In addition to sequencing systems, Pacific Biosciences provides consumables like SMRT cells and various reagent kits essential for template preparation, binding, and sequencing. Its diverse customer base includes research institutions, commercial laboratories, genome centers, and various sectors such as pharmaceuticals and agriculture. The company markets its products through a direct sales force in North America and Europe, along with distribution partners in Asia, the Middle East, and Latin America. Founded in 2000, Pacific Biosciences is headquartered in Menlo Park, California.
Pacific Biosciences designs, develops, and manufactures advanced sequencing systems that address complex genetic challenges. Utilizing single molecule real-time (SMRT) sequencing technology, it enables the real-time detection of biological processes at the single-molecule level. The company offers products such as the PacBio RS II and Sequel Systems, which are capable of conducting, monitoring, and analyzing biochemical reactions in real time. In addition to sequencing systems, Pacific Biosciences provides consumables like SMRT cells and various reagent kits essential for template preparation, binding, and sequencing. Its diverse customer base includes research institutions, commercial laboratories, genome centers, and various sectors such as pharmaceuticals and agriculture. The company markets its products through a direct sales force in North America and Europe, along with distribution partners in Asia, the Middle East, and Latin America. Founded in 2000, Pacific Biosciences is headquartered in Menlo Park, California.