Alexandria Venture Investments

Meiogenix is a biotechnology company developing breeding technologies to uncover unexplored genetic diversity in organisms, expanding natural biodiversity and enabling the development of products to address global food, climate, sustainability, and industrial challenges. It uses chromosome-editing-based approaches to modulate the frequency of homologous recombination in eukaryotic cells, enabling natural breeding and delivering new products for farmers and industry.

March Biosciences is a clinical-stage biotechnology company specializing in cell therapies for hematological cancers. It focuses on developing CAR-T cell therapies, with its lead product, MB-105, targeting CD5 in refractory T-cell lymphoma and leukemia. The company's approach involves biologically informed target selection, simplified CAR-T engineering, and a streamlined manufacturing process to ensure cell functionality and consistency.

Judo Bio is a biotechnology company developing precision therapeutics that target specific cell populations to address genetic diseases. Its innovative receptor targeting approach aims to benefit patients with both common and rare diseases.

858 Therapeutics is a biotechnology company specializing in the discovery of small molecule therapeutics for novel targets in oncology and immunology. The company focuses on innovative approaches such as synthetic lethality, innate immunity, and RNA modulation, with its lead program being a PARG inhibitor currently in early clinical development.

OverT Bio is a biotechnology company focused on developing cell therapies for cancer treatment. The company specializes in identifying and creating novel genetic modifications that empower immune cells to navigate and overcome the obstacles posed by tumors. Through its innovative approach, OverT Bio aims to enhance the effectiveness and durability of immunotherapies, contributing to advancements in cancer identification and treatment within the healthcare sector.

5Metis is a pure-play discovery company focused on crop health. It emerged from the combination of the Boragen and AgriMetis platforms, integrating boron-based small-molecule discovery with synthetic biology to identify new modes of action for crop protection and health. The company aims to deliver novel modes of action not currently available in the agrichemical industry, enabling farmers to improve crop protection and overall crop health through targeted solutions.

Clasp Therapeutics develops precision oncology platforms to identify neoantigens from driver mutations in hard-to-treat cancers. Its technology enables the creation of targeted therapies that selectively bind to HLA-presented peptides, allowing for precise destruction of cancer cells across various tumor types.

Elo Life Systems is a biotechnology company dedicated to enhancing human health through innovative food solutions. It aims to bridge gaps between food, agriculture, and health by partnering with stakeholders across the food systems value chain. Elo employs precision breeding technologies, genomics, data analytics, machine learning, and gene editing capabilities to develop healthier, more sustainable food options.

Remix Therapeutics Inc. is a biotechnology company focused on the research and development of innovative small-molecule therapies aimed at reprogramming ribonucleic acid (RNA) processing to treat various diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes its proprietary biology platform to discover and develop RNA processing modulators that target previously undruggable disease drivers. Through its REMseq platform, Remix Therapeutics validates therapeutic targets and optimizes chemical compounds, facilitating the identification of patterns in RNA processing. This approach allows for the modulation of gene expression, enabling healthcare practitioners to address the underlying causes of diseases by correcting, enhancing, or eliminating gene messages at their source.

Tome Biosciences is a biotechnology company specializing in programmable gene insertion technology. Utilizing CRISPR, the company develops methods to insert genetic sequences of any size at various locations within any genome. This innovative approach aims to enhance the ability to detect and eliminate deoxyribonucleic acid from similar bacteriophages during infections. By advancing cell and genome engineering, Tome Biosciences seeks to provide effective therapeutic solutions for patients, paving the way for significant advancements in genetic medicine.

Triveni Bio is a biotechnology company specializing in the development of novel antibody therapies for immunological and inflammatory disorders. The company employs a genetics-driven precision medicine approach to identify patients most likely to respond to its treatments, aiming to accelerate drug development and improve the effectiveness and safety of therapies.

Function Oncology is a precision medicine company focused on transforming cancer treatment through its innovative CRISPR-powered personalized functional genomics platform. This platform enables the identification of therapeutic opportunities tailored to individual patients, allowing for a deeper understanding of cancer at a patient-specific level. By leveraging advanced genomic techniques, Function Oncology aims to develop targeted cancer therapies that address the unique characteristics of each patient's disease, ultimately advancing the future of cancer care.

Ring Therapeutics is a biotechnology company developing gene therapies using Anellovector, a redosable and targetable DNA therapy platform based on the biology of the human commensal virome. The platform aims to address limitations of current DNA therapies, including limited tissue access, inability to redose, potential genomic integration, and tolerability concerns. Founded in 2017 and based in Cambridge, Massachusetts, Ring Therapeutics was established by Flagship Pioneering to pursue a multi-product platform. It focuses on discovering and developing Anellovector therapeutics to unlock the full potential of gene therapy, moving beyond gene replacement to a broader set of modalities and mechanisms. The approach targets disease sites with tissue and cellular specificity, aiming to treat conditions across genetic disorders, ophthalmology, oncology, and metabolic disorders and to expand therapeutic options for previously inaccessible diseases.

Tiamat Sciences is a start-up focused on producing affordable, animal-free growth factors and biomolecules through innovative plant-based bioreactors. Utilizing proprietary technology, the company manufactures a diverse array of high-value proteins, including antigens and growth factors, achieving significant cost reductions—up to 10 times lower than traditional methods, with potential for further reductions at scale. This technology not only enhances production efficiency but also supports ecological sustainability by providing plant-based alternatives to animal-derived materials in the pharmaceutical industry. Tiamat's approach increases the safety and speed of production, positioning it as a strategic partner for scaling operations in burgeoning sectors such as cellular agriculture and gene therapy. The low capital investment required for facility setup makes Tiamat an attractive option in a market poised for substantial growth.

Paratus Sciences focuses on advancing human health and health security by studying the biology of bats. It integrates cell biology, genomics, informatics, and large-scale data inputs to compare disease resistance patterns in bats with those in humans, aiming to develop therapeutics that mimic bats' adaptations.

Cajal Neuroscience is a biotechnology company dedicated to discovering novel targets and therapeutics for neurodegeneration. It integrates human genetics, functional genomics, and advanced microscopy to accelerate drug discovery in this field.

Rezo Therapeutics is a biotechnology company that focuses on pioneering the integrated mapping of disease networks to develop precision therapeutics. Utilizing its proprietary Sequence to Systems to Drugs (SSD) platform, Rezo combines proteomics, genetics, structural biology, chemistry, and bioinformatics to create comprehensive maps of molecular disease networks. This innovative approach enables oncologists and researchers to identify novel targets and therapies with greater clarity and precision than traditional drug discovery methods. Initially concentrating on oncology, Rezo Therapeutics plans to expand its therapeutic focus through collaborations and partnerships, aiming to address a wide range of diseases.

Haystack Oncology is an oncology company that specializes in advanced liquid biopsy diagnostic technology aimed at improving cancer treatment strategies and patient outcomes. The company utilizes state-of-the-art circulating tumor DNA detection methods to enhance the diagnosis of minimal residual disease, allowing for more informed therapy decision-making. By analyzing tumor-specific markers in blood samples, Haystack Oncology's technology provides high sensitivity for early detection of recurrent disease, thereby enabling healthcare teams to effectively manage cancer treatment. The company's innovative approach seeks to transform how cancer is monitored and treated, ultimately benefiting patients through improved diagnostic accuracy and treatment effectiveness.

Novome Biotechnologies, Inc. is a biotechnology company specializing in the engineering of gut bacteria to create innovative therapies for chronic diseases. Established in 2015 and located in South San Francisco, California, Novome has developed a pioneering platform for the controlled colonization of the gut with genetically engineered microbial medicines (GEMMs). This platform allows for the precise delivery of therapeutic activities aimed at treating conditions such as hyperoxaluria, with a focus on creating a bacterial strain that can degrade oxalate to help prevent kidney stone formation. The company is also expanding its synthetic biology capabilities to explore additional therapeutic indications, aiming to improve long-term health outcomes for patients through its living therapeutics.

Cartography Biosciences specializes in developing precision cancer immunotherapies. It employs single-cell analysis to identify novel targets for Chimeric Antigen Receptor T-cell (CAR-T) therapies, aiming to enhance safety and efficacy.

Pleno is a biotechnology company based in San Diego, California, founded in 2017. The company specializes in accelerating disease diagnosis and treatment through its innovative multi-omic platform. This technology is designed for biological target detection in clinical testing and biomedical research, offering capabilities for monitoring minimal residual disease and early multi-cancer screening. Pleno's platform detects a wide range of analytical targets, including DNA, RNA, and protein biomarkers, with exceptional speed and precision. By utilizing advanced microfluidics, the company automates all aspects of sample preparation and analysis, thereby enhancing healthcare providers' access to diverse health monitoring technologies.

Remix Therapeutics Inc. is a biotechnology company focused on the research and development of innovative small-molecule therapies aimed at reprogramming ribonucleic acid (RNA) processing to treat various diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes its proprietary biology platform to discover and develop RNA processing modulators that target previously undruggable disease drivers. Through its REMseq platform, Remix Therapeutics validates therapeutic targets and optimizes chemical compounds, facilitating the identification of patterns in RNA processing. This approach allows for the modulation of gene expression, enabling healthcare practitioners to address the underlying causes of diseases by correcting, enhancing, or eliminating gene messages at their source.

Affini-T Therapeutics is a biotechnology company developing T-cell therapies for patients suffering from cancer. Affini-T uses a validated platform to select high-affinity T cell receptors specific to oncogenic mutations to pioneer cutting-edge solid tumor therapies. Affini-T leverages gene editing and synthetic biology to engineer novel T-cell therapies designed to eradicate tumors.

Eclipse Bioinnovations is an RNA genomics company focused on advancing RNA medicines and scientific discovery through platform technologies that map RNA binding proteins, RNA interactions, and transcript features. Building on UC San Diego research, it offers eCLIP-based tools such as eCLIP-RBP, miR-eCLIP, and m6A-eCLIP, End-Seq for 5' and 3' ends, and FLI-Seq to streamline CRISPR library prep. Products are provided as services and kits for biopharma companies and academic researchers, with customized solutions for clients. The company also develops cell-free genomics technologies that improve crosslinking and immunoprecipitation to identify RNA binding protein targets, accelerating research and drug development across diseases.

Ambys Medicines, Inc. is a biotechnology company based in Redwood City, California, focused on discovering and developing innovative therapies for severe liver diseases. Founded in 2016, Ambys aims to transform the lives of individuals suffering from liver conditions by creating advanced gene, drug, and cell therapies. The company is dedicated to restoring liver function, halting the progression of liver disease, and addressing the serious complications associated with liver failure. Through its regenerative medical therapies, Ambys seeks to provide relief to patients with chronic liver diseases, ultimately improving their quality of life.

Meiogenix is a biotechnology company developing breeding technologies to uncover unexplored genetic diversity in organisms, expanding natural biodiversity and enabling the development of products to address global food, climate, sustainability, and industrial challenges. It uses chromosome-editing-based approaches to modulate the frequency of homologous recombination in eukaryotic cells, enabling natural breeding and delivering new products for farmers and industry.

GeneCentric Diagnostics is engaged in the development and commercialization of molecular diagnostic tests aimed at assisting oncologists and patients in cancer treatment. The company has established two primary platform technologies: The Lung Subtype Platform (LSP), which classifies lung cancer patients into specific subtypes to guide therapeutic choices, and the Hypoxia Signature, designed to identify patients likely to benefit from anti-angiogenesis therapies. The LSP has been licensed to Laboratory Corporation of America Holdings and is available as HistoPlusSM: Lung Cancer through its Integrated Oncology division. GeneCentric's innovative partnership model facilitates the translation of significant cancer research into diagnostics that are clinically adopted by pathologists and clinicians. Incorporated in 2011 and located in Durham, North Carolina, GeneCentric aims to enhance treatment outcomes by enabling more precise targeting of therapeutic compounds based on tumor biology.

Acrigen Biosciences is focused on advancing gene editing technologies to enhance the safety and efficiency of CRISPR-Cas based systems. Founded in 2019 and headquartered in Berkeley, California, the company develops precision gene editing solutions aimed at producing effective and safe in vivo drug therapies for various diseases. Acrigen's innovations include Acro proteins that regulate the CRISPR-Cas gene editing process, along with a bioinformatics discovery software platform designed to identify suitable CRISPR systems for human therapeutic applications. By improving anti-CRISPR proteins and optimizing genetic therapies, Acrigen aims to contribute significantly to the field of medicine and the treatment of debilitating conditions.

Kytopen Corp., founded in 2017, develops proprietary Flowfect™ technology for non-viral delivery of molecules into hard-to-transfect immune cells. Its platform combines microfluidics and automation to facilitate efficient cell engineering.

ROME Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing innovative therapies for cancer and autoimmune diseases. Founded in 2019, the company utilizes the repeatome, which encompasses extensive segments of genetic material previously regarded as "junk DNA," to identify new drug targets and advance multiple discovery programs. ROME Therapeutics has assembled a team of experts in oncology, immunology, virology, and machine learning to drive its research and development efforts, aiming to harness this uncharted area of biology for effective treatments. Through its work, ROME seeks to provide healthcare professionals with powerful new options for addressing complex medical challenges.

Thryv Therapeutics is a precision medicine company focused on developing innovative therapeutics for Long QT Syndrome and other arrhythmias. The company employs a precision medicine approach to address both genetic and drug-induced long QT syndrome, as well as atrial fibrillation and treatment-resistant cancers. Thryv Therapeutics specializes in creating potent and selective inhibitors of Serum Glucocorticoid Inducible Kinase, aiming to provide therapies that can significantly reduce the risk of sudden cardiac death and enhance the quality of life for patients affected by these serious conditions.

Deep Genomics is a Toronto-based company founded in 2014. It specializes in developing individualized genetic medicines using artificial intelligence systems to accelerate drug discovery and development processes, including target discovery, lead optimization, toxicity assessment, and innovative trial design. The company focuses on therapies for rare metabolic, ophthalmologic, and neurodegenerative disorders, utilizing oligonucleotide therapies that target genetic determinants of disease at the RNA or DNA level.

Mnemo Therapeutics is a biotechnology company developing immune-based therapies, including cell therapies. Its EnfiniT platform uses epigenetic variation and CAR-T precision genetic engineering technology to target tumorous epitopes across various cancers.

miRecule is a biotechnology company developing targeted RNA therapies for cancer and muscular dystrophy. It uses its proprietary DREAmiR platform to analyze genomic data from thousands of patients, identifying genetic abnormalities and creating tailored RNA therapeutics to directly address these issues.

Inari is a biotechnology company focused on revolutionizing agriculture and its societal and environmental impact. The company utilizes advanced technologies, including predictive design and multiplex gene editing, to maximize the potential of seeds. Its unique SEEDesign platform distinguishes Inari by enabling the enhancement of various crops across different geographies, facilitating the development of innovative agricultural products. By embracing diversity, Inari fosters innovation and aims to contribute to the creation of a new food system.

TwinStrand Biosciences, established in 2015 and based in Bellevue, Washington, specializes in advancing DNA sequencing technology. The company's core innovation, Duplex Sequencing, enhances sequencing accuracy by over 10,000 times, enabling the detection of previously invisible mutations. This breakthrough technology has wide-ranging applications, including oncology, infectious disease diagnosis, organ transplant monitoring, genetic health, and forensics.

Molecular Assemblies, Inc. is a San Diego-based company that specializes in enzymatic DNA synthesis technology, aimed at creating DNA-based products across various industries. Founded in 2013, the company has developed a proprietary method that allows for the reliable, affordable, and sustainable production of long, high-quality, sequence-specific DNA. This innovative technology is designed to support advancements in industrial synthetic biology, personalized therapeutics, precision diagnostics, and has potential applications in information storage and nanotechnology. With a strong executive and scientific team, as well as a robust intellectual property portfolio, Molecular Assemblies is positioned to enhance the capabilities of researchers and contribute to the evolution of DNA-based applications.

SeQure DX is a biotechnology company specializing in genomics diagnostics for CRISPR-based gene editing therapies. It develops technologies that identify potential off-target sites prior to therapy initiation, ensuring comprehensive confirmation of actual edits. This enables safer and more effective gene editing therapeutics development, benefiting both researchers and patients.

Ensoma is a biotechnology company focused on advancing genomic medicine through its innovative in vivo approach, utilizing proprietary Engenious vectors. These vectors are engineered to deliver a wide array of gene modification technologies directly to blood and immune cells, including T cells, B cells, and myeloid cells, without the necessity for stem cell collection or prior myeloablative conditioning, which can pose risks to patients. This unique method allows for the administration of therapies via a single injection, enhancing accessibility and convenience in various healthcare settings, even where resources may be limited. Ensoma aims to expand the curative potential of genomic medicine, making it more accessible to patients in need.

Vera Therapeutics, Inc. is a biotechnology company based in South San Francisco, California, founded in 2016 and formerly known as Trucode Gene Repair, Inc. The company focuses on developing innovative biologic therapeutics with the potential to transform treatment for patients suffering from serious immunological diseases and genetic disorders. Its lead product candidate, atacicept, is a fusion protein administered as a subcutaneous injection once weekly, designed to inhibit factors that stimulate the production of autoantibodies associated with autoimmune diseases. Additionally, Vera Therapeutics is utilizing gene-editing technology aimed at addressing conditions such as sickle cell disease and cystic fibrosis, striving to enhance gene detection and regulation for improved patient outcomes.

Lexeo Therapeutics is a fully integrated biotechnology company focused on developing adeno-associated virus (AAV)-mediated gene therapies. Its lead programs target both rare and non-rare monogenic diseases, with a preclinical pipeline that spans monogenic, hereditary, and acquired conditions across populations with unmet medical need. The company aims to advance clinical programs toward commercialization while maintaining an ongoing research collaboration with Weill Cornell Medicine’s Department of Genetic Medicine to inform and accelerate its preclinical and clinical work. By leveraging AAV-based approaches and collaborations, Lexeo Therapeutics seeks to transform treatment options for patients with genetic and complex diseases.

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.

Remix Therapeutics Inc. is a biotechnology company focused on the research and development of innovative small-molecule therapies aimed at reprogramming ribonucleic acid (RNA) processing to treat various diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes its proprietary biology platform to discover and develop RNA processing modulators that target previously undruggable disease drivers. Through its REMseq platform, Remix Therapeutics validates therapeutic targets and optimizes chemical compounds, facilitating the identification of patterns in RNA processing. This approach allows for the modulation of gene expression, enabling healthcare practitioners to address the underlying causes of diseases by correcting, enhancing, or eliminating gene messages at their source.

Sonoma Biotherapeutics, Inc. is focused on developing adoptive T regulatory (Treg) cell therapies aimed at treating autoimmune and degenerative diseases. Established in 2019 and based in South San Francisco, California, with an additional office in Seattle, the company employs next-generation genome editing and target-specific cell therapy techniques to innovate in the field of Treg cell therapeutics. The firm aims to create disease-modifying and potentially curative therapies that promote self-tolerance and inhibit harmful inflammatory responses associated with conditions such as rheumatoid arthritis, inflammatory bowel disease, multiple sclerosis, amyotrophic lateral sclerosis, and Alzheimer's disease. Founded by experts in Treg biology and cell therapy, Sonoma Biotherapeutics integrates advanced methodologies to enhance its therapeutic offerings.

Korro Bio Inc., established in 2018 and headquartered in Cambridge, Massachusetts, specializes in developing nucleic acid-based therapeutics targeting specific sequences in genetic code to treat rare diseases. The company's proprietary platform enables efficient and selective RNA editing, leveraging natural processes common to all multicellular organisms. Korro Bio aims to advance this technology to effect changes in protein structure and function across multiple tissues, expanding the reach of genetic medicines with additional precision and tunability by editing RNA instead of DNA.

Element Biosciences develops innovative genetic analysis tools for research and diagnostic markets. Founded in 2017, the company specializes in a disruptive DNA sequencing technology that offers high-quality data, workflow flexibility, and affordable access to next-generation sequencing.

Affinia Therapeutics develops adeno-associated virus (AAV) gene therapies for muscular and central nervous system diseases. The company uses an AAV vector design platform built on synthetic and systems biology, high-throughput screening, and tissue- and single-cell-resolution analyses to create vectors with improved properties. It leverages sequencing, artificial intelligence, and structural modeling to enable rational design of therapies. Headquartered in Waltham, Massachusetts, Affinia Therapeutics was founded in 2019 and targets diseases with significant unmet needs in muscle and CNS.

Redpin Therapeutics, Inc. is a preclinical stage gene therapy company founded in 2017 and based in New York, New York. The company specializes in developing a proprietary chemogenetics platform for targeted cell therapies aimed at addressing intractable diseases of the nervous system. Redpin's innovative approach utilizes ion channels as neuromodulation tools, allowing for selective activation or inhibition of disease-causing neurons while preserving normal functioning cells. By controlling these neuronal circuits, Redpin enables physicians to precisely target dysfunctional neural activity associated with specific conditions. The company's technology integrates principles from synthetic biology, gene therapy, and traditional pharmacotherapy to advance its mission of addressing complex neurological disorders.

Kallyope Inc. is a biotechnology company based in New York City that focuses on understanding the gut-brain axis to develop innovative therapeutics and consumer products aimed at enhancing human health. Established in 2014, Kallyope employs advanced technologies, including sequencing, computational biology, neural imaging, and human genetics, to explore gut-brain biology. The company aims to address significant unmet medical needs, such as diabetes, obesity, gut disorders, inflammatory diseases, migraines, and allergies, by creating effective and user-friendly oral agents. Kallyope's team consists of experienced scientists from leading institutions, working alongside a skilled team with a strong track record in drug discovery and development. Through its research, Kallyope seeks to deliver transformative solutions that improve overall health outcomes.

TwinStrand Biosciences, established in 2015 and based in Bellevue, Washington, specializes in advancing DNA sequencing technology. The company's core innovation, Duplex Sequencing, enhances sequencing accuracy by over 10,000 times, enabling the detection of previously invisible mutations. This breakthrough technology has wide-ranging applications, including oncology, infectious disease diagnosis, organ transplant monitoring, genetic health, and forensics.

Novome Biotechnologies, Inc. is a biotechnology company specializing in the engineering of gut bacteria to create innovative therapies for chronic diseases. Established in 2015 and located in South San Francisco, California, Novome has developed a pioneering platform for the controlled colonization of the gut with genetically engineered microbial medicines (GEMMs). This platform allows for the precise delivery of therapeutic activities aimed at treating conditions such as hyperoxaluria, with a focus on creating a bacterial strain that can degrade oxalate to help prevent kidney stone formation. The company is also expanding its synthetic biology capabilities to explore additional therapeutic indications, aiming to improve long-term health outcomes for patients through its living therapeutics.

Triplet Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for triplet repeat expansion disorders, such as Huntington's disease, myotonic dystrophy, and spinocerebellar ataxias. Founded in 2018 and based in Cambridge, Massachusetts, the company aims to address these conditions at their source by utilizing a unique approach that involves a single oligonucleotide targeting the DNA Damage Response pathway. This strategy is designed to provide effective treatments for multiple repeat expansion disorders, enabling healthcare professionals to offer advanced biotherapeutic options to their patients. Through its research and development efforts, Triplet Therapeutics is committed to transforming the landscape of treatment for these challenging genetic disorders.

Aspen Neuroscience develops autologous neuron replacement therapies based on induced pluripotent stem cells to treat neurological conditions, starting with Parkinson's disease. The company uses patient-specific restorative cell therapies by combining pluripotent stem cell biology with genomic approaches and artificial intelligence to address both sporadic and familial forms of Parkinson's disease, focusing on autologous neuron replacement to modify disease progression. Based in San Diego, California, the company is a development-stage biotechnology company pursuing expansion of its iPSC-based platform to other brain disorders.

Jasper Therapeutics, Inc. is a clinical-stage biotechnology company based in Menlo Park, California, that specializes in developing therapeutic agents for hematopoietic stem cell transplants and gene therapies. Founded in 2018, the company aims to enhance the safety and efficacy of conditioning agents to broaden the application of curative therapies. Jasper's lead product, JSP191, is a humanized monoclonal antibody currently in clinical development, designed to act as a conditioning agent by selectively clearing hematopoietic stem cells from bone marrow in patients undergoing stem cell transplants. The company also utilizes an engineered hematopoietic stem cells platform to address the limitations of transplant grafts, thereby increasing the accessibility of both allogeneic and autologous gene-edited hematopoietic stem cell therapies for a larger patient population.

Immunitas Therapeutics, Inc. is a biotechnology company based in Cambridge, Massachusetts, focused on developing targeted therapeutics for patients with complex cancers. Founded in 2019, the company utilizes a single-cell sequencing platform to analyze the biology of immune cells within human tumors. This innovative approach allows Immunitas to identify novel drug targets and develop key biomarkers that guide patient selection for their therapies. By emphasizing research rooted in human biology, Immunitas aims to bridge the gap between laboratory discoveries and clinical applications. The company is advancing multiple programs toward early human studies, leveraging its expertise in antibody discovery and engineering to create effective treatments that modulate identified targets.

Genetron Health is a leading precision medicine company specializing in oncology, with a strong focus on cancer molecular profiling. The company leverages advanced technologies in molecular biology and data science to enhance cancer treatment. Genetron Health offers comprehensive health management products covering all aspects of full-cycle cancer clinical treatment, including risk assessment, early screening, molecular pathology diagnosis, medication guidance, and prognosis monitoring. They provide reliable molecular clinical services and professional cancer genetic risk assessments for patients and high-risk individuals. With global genomics sequencing and bioinformatics platforms, research and development centers in North Carolina and Beijing, and clinical laboratories across major Chinese cities, Genetron Health aims to expand its sales network throughout China. The company is dedicated to serving both Chinese and global cancer patients with its expertise in cancer genomics and efficient clinical translation.

miRecule is a biotechnology company developing targeted RNA therapies for cancer and muscular dystrophy. It uses its proprietary DREAmiR platform to analyze genomic data from thousands of patients, identifying genetic abnormalities and creating tailored RNA therapeutics to directly address these issues.

Variant Bio Inc. is a biotechnology company focused on developing therapeutics through the exploration of human genetic diversity. Founded in 2018 and headquartered in Seattle, Washington, the company seeks to identify individuals and populations exhibiting extreme outlier traits relevant to various medical conditions. By employing advanced sequencing technologies, statistical genetics, and machine learning, Variant Bio analyzes the genetic underpinnings of these traits to uncover new therapeutic targets. The company's mission is to enhance global health by addressing unmet medical needs, particularly in areas such as neurodegenerative, autoimmune, and cardiometabolic diseases.

Molecular Assemblies, Inc. is a San Diego-based company that specializes in enzymatic DNA synthesis technology, aimed at creating DNA-based products across various industries. Founded in 2013, the company has developed a proprietary method that allows for the reliable, affordable, and sustainable production of long, high-quality, sequence-specific DNA. This innovative technology is designed to support advancements in industrial synthetic biology, personalized therapeutics, precision diagnostics, and has potential applications in information storage and nanotechnology. With a strong executive and scientific team, as well as a robust intellectual property portfolio, Molecular Assemblies is positioned to enhance the capabilities of researchers and contribute to the evolution of DNA-based applications.

Boundless Bio develops innovative cancer therapeutics focused on understanding and treating previously untreatable cancers. It specializes in targeting extrachromosomal DNA (ecDNA) biology, a key driver of aggressive cancers.

X-Vax Technology is a biotechnology company established in 2015 and headquartered in Jupiter, Florida. Its primary focus is the preclinical and clinical development of vaccines targeting mucosal infections caused by pathogens, including herpes, influenza, tuberculosis, and HIV, which affect millions globally. X-Vax is known for its development of a herpes vaccine candidate based on a genetically altered HSV-2 virus, specifically designed to elicit a robust immune response by inducing antibodies that lead to the killing of infected cells. This innovative approach utilizes Fc receptor activating antibodies that facilitate antibody-dependent cell-mediated killing, aiming to provide effective protection against herpes types 1 and 2. Through its pioneering research, X-Vax intends to advance the field of vaccines and enhance immune responses against significant viral infections.

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.

Cardea Bio, Inc. is a biotechnology company based in San Diego, California, focused on developing and commercializing bioelectronics for research and diagnostics. Established in 2013, Cardea utilizes proprietary graphene-based digital biosensors to create advanced biosensor hardware, software, and molecular infrastructure, enabling precision healthcare through improved diagnostic capabilities. The company's product offerings include the AGILE R100, an innovative assay for drug discovery, and the AGILE R200, an automated high-throughput screening solution. Cardea's biosensors convert biomolecular interactions into digital data, allowing for real-time biological signal analysis and access to streaming multi-omics data. Their solutions cater to various applications in life sciences and healthcare, such as small molecule validation, antibody characterization, and biomolecular analysis.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

Gencove specializes in a software platform for efficient and cost-effective genome sequencing using low-pass sequencing methods. This enables high-throughput genomics research and diagnostics across various species.

Maze Therapeutics is a biotechnology company focused on developing medicines that mimic the effects of rare, protective genetic variants to address unmet medical needs. Utilizing its Compass platform, the company identifies genetic variants associated with diseases and maps them to the biological pathways driving these conditions in specific patient populations.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

Immusoft is a biotechnology company that develops autologous cell therapies using its Immune System Programming (ISP) technology. Founded in 2009 and headquartered in Seattle, Washington, the company reprograms patients' cells to produce gene-encoded medicines for treating various diseases.

Kallyope Inc. is a biotechnology company based in New York City that focuses on understanding the gut-brain axis to develop innovative therapeutics and consumer products aimed at enhancing human health. Established in 2014, Kallyope employs advanced technologies, including sequencing, computational biology, neural imaging, and human genetics, to explore gut-brain biology. The company aims to address significant unmet medical needs, such as diabetes, obesity, gut disorders, inflammatory diseases, migraines, and allergies, by creating effective and user-friendly oral agents. Kallyope's team consists of experienced scientists from leading institutions, working alongside a skilled team with a strong track record in drug discovery and development. Through its research, Kallyope seeks to deliver transformative solutions that improve overall health outcomes.

Ribometrix is a biotechnology company that develops platform therapeutics to discover and advance small molecule drugs that target functional three-dimensional RNA structures to treat human diseases. Founded in 2014 and based in Durham, North Carolina, it focuses on leveraging RNA structure analysis and proprietary tools, along with artificial intelligence, to identify compounds that modulate RNA biology and reduce disease-associated protein production.

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.

Inspirna is a clinical-stage biopharmaceutical company focused on developing first-in-class cancer therapies that target RNA-regulated drivers of tumor growth. Using RNA biology-based discovery platforms, including miRNA-DRIVEr and RNA-DRIVEr, it identifies novel cancer targets and advances them with oral small-molecule medicines and antibody-drug conjugates. Its lead programs include RGX-202 for RAS-mutant colorectal cancer and RGX-104 for small cell and non-small cell lung cancers, currently in early-phase trials (Phase 1b/2). The company aims to translate RNA-centric targets into treatments for patients with cancers that lack effective options.

Epic Sciences is a diagnostics company focused on cancer management through blood-based tests that identify and characterize circulating tumor cells from a minimally invasive sample. Its platform profiles single-cell phenotype and genotype, including biomarker expression levels, subcellular localization, morphologic features, and genotypic metrics, and supports next-generation sequencing, FISH, and single-cell genomics to detect and characterize CTCs. The company develops tests such as the Oncotype DX AR-V7 Nucleus Detect for metastatic castration-resistant prostate cancer and offers biopharma solutions. It collaborates with biotechnology and pharmaceutical companies and cancer centers worldwide to provide real-time biopsy material and data to guide personalized medicine. By enabling analysis of rare cells before and after treatment, Epic Sciences aims to improve cancer management. The company was founded in 2008 and is headquartered in San Diego, California.

Inari is a biotechnology company focused on revolutionizing agriculture and its societal and environmental impact. The company utilizes advanced technologies, including predictive design and multiplex gene editing, to maximize the potential of seeds. Its unique SEEDesign platform distinguishes Inari by enabling the enhancement of various crops across different geographies, facilitating the development of innovative agricultural products. By embracing diversity, Inari fosters innovation and aims to contribute to the creation of a new food system.

ORIG3N, Inc. is a biotechnology company focused on developing innovative treatments for rare genetically inherited diseases, particularly targeting conditions related to the heart, liver, and neurodegeneration. Founded in 2014 and based in Boston, Massachusetts, ORIG3N utilizes induced pluripotent stem cell technology that allows for the differentiation of cells into any type found in the body. This technology is employed to test drug efficacy, screen for medication toxicity, and create personalized treatment pathways. The company offers a range of products, including LifeProfiles, which provides genetic insights related to fitness, metabolism, and exercise recovery; the Run DNA test for optimizing running performance; AURA, which assesses skin health; and Life Capsule, a repository for blood cells aimed at regenerative medicine development.

Skyhawk Therapeutics, Inc. is a biotechnology company focused on the discovery and development of small molecule therapeutics that aim to correct RNA expression. The company's proprietary technology facilitates the rational design of these small molecules to target specific binding pocket regions on RNA, employing both sequence and structural specificity at critical moments during the RNA splicing process. This innovative approach allows for the targeting of diseases associated with RNA mis-splicing, particularly those characterized by exon skipping. Skyhawk Therapeutics is committed to developing treatments for various conditions, including cancer, neurological diseases, and rare disorders. Founded in 2015 and headquartered in Waltham, Massachusetts, the company has evolved from its initial identity as Rare Genetix, Inc., adopting its current name in May 2018.

Cibus is a biotechnology company specializing in precision gene editing for agriculture, aimed at making farming more sustainable and profitable. The company has developed patented breeding technologies that facilitate precise and predictable modifications in plants without the integration of foreign genetic material. Its core innovation, the Rapid Trait Development System, combines crop-specific cell biology platforms with advanced gene editing techniques. Cibus focuses on creating plant traits that address specific agricultural challenges, including those related to productivity, disease resistance, insect control, weed management, nutrient use, and climate adaptability. Through these efforts, Cibus seeks to enhance the efficiency and sustainability of agricultural practices.

StrideBio, Inc. is a gene therapy company based in Durham, North Carolina, focused on developing innovative genetic medicines for rare diseases. Founded in 2015, the company utilizes a proprietary structure-inspired adeno-associated virus (AAV) vector engineering platform to create unique and differentiated vectors. These vectors are designed to improve upon naturally occurring AAV serotypes, addressing limitations of first-generation gene therapies. StrideBio's technologies facilitate gene addition, silencing, and editing, providing potential curative options for patients with severe medical conditions. By combining structural insights with accelerated evolution, StrideBio aims to produce novel AAV capsids capable of evading neutralizing antibodies, ultimately enhancing treatment outcomes for those affected by rare diseases.

Celsius Therapeutics, Inc. is a biotechnology company focused on developing precision medicines for patients suffering from cancer and autoimmune diseases. Founded in 2018 and based in Cambridge, Massachusetts, the company employs a multidisciplinary approach, integrating the expertise of scientists, computational biologists, and clinicians. Celsius Therapeutics utilizes advanced techniques such as single-cell genomic analysis and machine learning to identify and develop innovative therapeutic solutions. By leveraging these cutting-edge technologies, the company aims to create life-changing medicines that address unmet medical needs.

QurAlis Corporation is a Cambridge, Massachusetts-based clinical-stage biotechnology company developing precision therapeutics for amyotrophic lateral sclerosis and other neurodegenerative diseases. It focuses on drugs that directly target disease-causing genetic alterations using proprietary platforms and biomarkers to identify and treat patients with specific genetic mutations, aiming to halt disease progression and improve outcomes.

Kallyope Inc. is a biotechnology company based in New York City that focuses on understanding the gut-brain axis to develop innovative therapeutics and consumer products aimed at enhancing human health. Established in 2014, Kallyope employs advanced technologies, including sequencing, computational biology, neural imaging, and human genetics, to explore gut-brain biology. The company aims to address significant unmet medical needs, such as diabetes, obesity, gut disorders, inflammatory diseases, migraines, and allergies, by creating effective and user-friendly oral agents. Kallyope's team consists of experienced scientists from leading institutions, working alongside a skilled team with a strong track record in drug discovery and development. Through its research, Kallyope seeks to deliver transformative solutions that improve overall health outcomes.

Skyhawk Therapeutics, Inc. is a biotechnology company focused on the discovery and development of small molecule therapeutics that aim to correct RNA expression. The company's proprietary technology facilitates the rational design of these small molecules to target specific binding pocket regions on RNA, employing both sequence and structural specificity at critical moments during the RNA splicing process. This innovative approach allows for the targeting of diseases associated with RNA mis-splicing, particularly those characterized by exon skipping. Skyhawk Therapeutics is committed to developing treatments for various conditions, including cancer, neurological diseases, and rare disorders. Founded in 2015 and headquartered in Waltham, Massachusetts, the company has evolved from its initial identity as Rare Genetix, Inc., adopting its current name in May 2018.

Expansion Therapeutics is a drug discovery and development company focused on creating small molecule medicines for RNA-mediated diseases. The company specializes in developing novel RNA-targeted drug candidates that aim to address a wide range of severe medical conditions. By leveraging its innovative technology, Expansion Therapeutics seeks to provide transformative oral treatment options, enhancing the therapeutic landscape for patients suffering from these complex diseases.

Founded in 2015 and based in Cambridge, Massachusetts, Obsidian Therapeutics develops next-generation controllable cell and gene therapies designed to extend adoptive immunotherapy to every cancer patient. Its innovative approach uses pharmacologic operating systems to precisely control protein activity in cells, enabling physicians to improve upon current cell therapies using simple, safe, orally-active marketed drugs.

Ribometrix is a biotechnology company that develops platform therapeutics to discover and advance small molecule drugs that target functional three-dimensional RNA structures to treat human diseases. Founded in 2014 and based in Durham, North Carolina, it focuses on leveraging RNA structure analysis and proprietary tools, along with artificial intelligence, to identify compounds that modulate RNA biology and reduce disease-associated protein production.

Cydan is an orphan drug accelerator focused on developing therapies for patients with rare genetic diseases. The company identifies and de-risks compounds that hold therapeutic and commercial potential, employing a rigorous process to generate data that supports both development pathways and the formation of new companies. With a team that boasts deep expertise in drug development and strong relationships within academia and patient advocacy, Cydan accelerates therapies through various stages, including clinical, regulatory, and commercial development. By facilitating the creation of effective treatments, Cydan aims to improve the lives of patients and families affected by rare diseases.

LifeMine Therapeutics, Inc. is a biotechnology company focused on the genomic discovery of new drug modalities derived from eukaryotic microbes, specifically fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, it employs an integrated drug discovery platform that combines genomics, artificial intelligence, and synthetic biology. LifeMine’s Avatar-Rx platform utilizes high-throughput microbiology, data science, genome engineering, and automation technologies to identify novel gene-encoded molecules (GEMs) with specific biological functions. The company emphasizes chemoinformatic-assisted drug optimization and advanced chemical synthesis, enabling the progression of new product candidates into development. Founded by experts in the field, LifeMine Therapeutics aims to innovate medicine through its unique approach to leveraging the fungal biosphere.

Gritstone bio is a biotechnology company focused on developing immunotherapies for cancer and infectious diseases. The company initially concentrated on tumor-specific neoantigens but has expanded its programs to include viral antigens found on the surface of infected cells. Gritstone bio's approach leverages the body's natural immune system recognition of targets on abnormal cells, which is applicable to both anti-tumor and anti-viral immunity. Their pipeline includes several product candidates for treating solid tumors, such as GRANITE, SLATE, and CORAL. The company operates in a field that has seen significant advancements with the development and commercialization of immunotherapy drugs like checkpoint inhibitors, transforming cancer treatment.

Egenesis is a biotechnology company focused on gene editing and genome engineering to develop human-compatible organs, tissues, and cells for transplantation. Its platform enables the creation of solid organs and therapeutic cells intended for transplantation, addressing the global organ shortage. The company pursues programs in kidney and islet cell transplantation and related regenerative approaches. It is based in Cambridge, Massachusetts, with an additional office in New York.

Applied Therapeutics is a biopharmaceutical company focused on developing transformative drugs targeting fatal and debilitating diseases with high unmet medical needs. Its pipeline includes novel product candidates such as AT-007 for rare metabolic diseases like galactosemia, AT-001 for diabetic cardiomyopathy, and AT-003 for diabetic retinopathy.

Synthego is a biotechnology company specializing in genome engineering. It offers integrated hardware, software, bioinformatics, chemistries, and molecular biology platforms to advance basic research and therapeutic development. Its products include engineered cells, CRISPR kits, and bioinformatics tools for gene knockout, target validation, and disease modeling. Synthego serves scientists globally, with sales through distributors in multiple countries and online.

ApoGen Biotechnologies, Inc. is a biotechnology company based in Seattle, Washington, established in 2014. The company specializes in developing innovative therapeutics aimed at addressing the challenges of drug resistance in cancer treatment. Recognizing that the mutation of cellular DNA is central to cancer progression, ApoGen focuses on therapies that target key drivers of genomic mutations and tumor evolution. By doing so, the company aims to slow or halt the ongoing mutations within cancer genomes, which are significant contributors to treatment failure, cancer recurrence, and metastasis. Through its advanced therapeutic approaches, ApoGen Biotechnologies seeks to improve outcomes for patients facing the complexities of cancer evolution and drug resistance.

Molecular Assemblies, Inc. is a San Diego-based company that specializes in enzymatic DNA synthesis technology, aimed at creating DNA-based products across various industries. Founded in 2013, the company has developed a proprietary method that allows for the reliable, affordable, and sustainable production of long, high-quality, sequence-specific DNA. This innovative technology is designed to support advancements in industrial synthetic biology, personalized therapeutics, precision diagnostics, and has potential applications in information storage and nanotechnology. With a strong executive and scientific team, as well as a robust intellectual property portfolio, Molecular Assemblies is positioned to enhance the capabilities of researchers and contribute to the evolution of DNA-based applications.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is primarily engaged in the clinical study of VTS-270 for the treatment of Niemann-Pick Disease Type C (NPC) and is also involved in the pre-clinical discovery and development of additional novel drugs for NPC and other lysosomal storage diseases. Vtesse operates as a subsidiary of Sucampo Pharmaceuticals, Inc. and is recognized as the first spin-out from Cydan Development, Inc., an orphan-drug accelerator. The company collaborates with the National Institutes of Health (NIH) to advance its clinical programs and is supported by a seasoned management team with extensive experience in drug development. Vtesse's initiatives aim not only to provide innovative therapies but also to offer resources and support for patients and families affected by NPC and related conditions.

Imara, Inc. is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, that specializes in developing and commercializing therapeutics for patients with rare genetic disorders of hemoglobin. Founded in 2016, the company is advancing IMR-687, an oral, once-daily therapeutic designed to treat sickle cell disease and beta-thalassemia. IMR-687 is a highly selective small molecule inhibitor of PDE9, engineered to have a multimodal mechanism of action that impacts various cell types, including red blood cells and white blood cells, as well as adhesion mediators. Through its innovative approach, Imara aims to provide disease-modifying treatments for individuals affected by hemoglobinopathies.

EpiBiome, Inc. is a precision microbiome engineering company based in South San Francisco, California, founded in 2013. The company is dedicated to developing therapies that address infectious diseases in humans and agriculture without relying on small-molecule antibiotics. EpiBiome focuses on phage therapy, which targets specific strains within the microbiome, effectively treating antibiotic-resistant infections. Its applications include treatment for mastitis in dairy cows and addressing infections caused by Escherichia coli in humans. By utilizing advanced sequencing and bioinformatic processes, EpiBiome aims to identify pathogenic bacteria and microbiome changes that contribute to disease, offering innovative alternatives to combat the rising threat of antibiotic resistance. As of July 2018, it operates as a subsidiary of Locus Biosciences, Inc.

Kallyope Inc. is a biotechnology company based in New York City that focuses on understanding the gut-brain axis to develop innovative therapeutics and consumer products aimed at enhancing human health. Established in 2014, Kallyope employs advanced technologies, including sequencing, computational biology, neural imaging, and human genetics, to explore gut-brain biology. The company aims to address significant unmet medical needs, such as diabetes, obesity, gut disorders, inflammatory diseases, migraines, and allergies, by creating effective and user-friendly oral agents. Kallyope's team consists of experienced scientists from leading institutions, working alongside a skilled team with a strong track record in drug discovery and development. Through its research, Kallyope seeks to deliver transformative solutions that improve overall health outcomes.

Biological Dynamics Inc. is a healthcare company based in San Diego, California, that specializes in developing a proprietary technology platform for the direct and selective capture of large necrotic particles from physiological solutions such as whole blood, serum, and plasma. This platform facilitates the detection of diseases, particularly cancers, in their earliest stages by enabling access to native-state biomarkers and nanoparticles for multiomics applications. The company offers various products and services, including the TR(ACE) Assay for monitoring treatment responses in cancer, tissue-based molecular and cfDNA liquid biopsy testing for personalized medicine, and a biomarkers program aimed at drug development for Alzheimer's disease. Additionally, Biological Dynamics provides clinical laboratory services, including molecular and next-generation sequencing, and supports applications in autoimmune diseases, transplant rejection, and traumatic brain injury. Founded in 2008, the company is dedicated to improving global health outcomes through innovative diagnostic solutions.

Calimmune is a clinical-stage gene therapy company focused on HIV, developing cell-based therapies to protect individuals with HIV from progression to AIDS. Its lead candidate, Cal-1, is a gene-based therapy designed to modify immune cells to reduce HIV entry and replication by lowering CCR5 and preventing viral fusion, with activity demonstrated in preclinical studies and evaluated in Phase I/II trials. The company is based in Tucson, Arizona, and aims to address HIV at various stages of infection and treatment history by delivering durable cellular therapies that strengthen the immune system.

Vtesse, Inc. is a biopharmaceutical company based in Gaithersburg, Maryland, focused on developing treatments for patients with rare diseases. The company is primarily engaged in the clinical study of VTS-270 for the treatment of Niemann-Pick Disease Type C (NPC) and is also involved in the pre-clinical discovery and development of additional novel drugs for NPC and other lysosomal storage diseases. Vtesse operates as a subsidiary of Sucampo Pharmaceuticals, Inc. and is recognized as the first spin-out from Cydan Development, Inc., an orphan-drug accelerator. The company collaborates with the National Institutes of Health (NIH) to advance its clinical programs and is supported by a seasoned management team with extensive experience in drug development. Vtesse's initiatives aim not only to provide innovative therapies but also to offer resources and support for patients and families affected by NPC and related conditions.