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City Therapeutics is a biopharmaceutical company specializing in the development of next-generation small-interfering RNAs (siRNAs) to enhance RNA interference (RNAi) therapies. The company's pipeline comprises innovative RNAi therapeutics targeting diverse disease indications. City Therapeutics aims to improve and expand RNAi technology to achieve better patient outcomes, led by a team of experienced scientists and industry professionals committed to advancing the field of RNA interference.

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.

GenePlanet is a European biotechnology company that focuses on genetic testing and research. It offers a range of preventive genetic tests aimed at consumers, as well as clinical genetic tests that assist healthcare professionals in therapeutic decision-making. Their tests are based on the latest scientific advancements and adhere to high-quality standards. By utilizing saliva-based DNA testing, GenePlanet provides individuals with personalized insights into their genetic predispositions, allowing them to make informed lifestyle adjustments to mitigate health risks. This approach encourages improved health, vitality, and overall well-being through tailored prevention programs.

Asimov, Inc. is a biotechnology company that specializes in the design and development of genetically engineered therapeutics. Established in 2017 and based in Cambridge, Massachusetts, Asimov focuses on creating tools for programming living cells to enhance the design and manufacturing processes of biologics and gene therapies. The company's platform integrates synthetic biology, computer-aided design, and machine learning, allowing for the engineering of genetic circuits. Asimov's multidisciplinary team employs machine-learning algorithms that connect large-scale datasets with biological models, facilitating advancements in molecular manufacturing and therapeutic applications. The company is dedicated to optimizing the production of protein biologics, viral vectors, and cell and gene therapies.

Castle Creek Biosciences, Inc. is a gene therapy company headquartered in Exton, Pennsylvania, that specializes in developing and commercializing personalized gene therapies for rare skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin to create localized therapies tailored to the individual’s unique biology. This innovative approach aims to address the underlying causes of diseases that currently have high unmet medical needs, ultimately improving the lives of patients suffering from these underserved conditions.

LifeMine Therapeutics, Inc. is a biotechnology company focused on discovering new drug modalities from eukaryotic microbes, particularly fungi. Founded in 2016 and headquartered in Cambridge, Massachusetts, with an additional office in New York, the company employs an integrated platform that combines genomics, artificial intelligence, and synthetic biology. This innovative Avatar-Rx platform utilizes high-throughput microbiology, data science, machine learning, genome engineering, and automation to explore the fungal biosphere for novel genetically encoded molecules with specific biological functions. By integrating chemoinformatic-assisted drug optimization and advanced chemical synthesis with biotransformation, LifeMine Therapeutics aims to advance a pipeline of new medicines into development.

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 SAS, established in 2014 and headquartered in Paris, France, specializes in synthetic biology tools. The company's core product, SYNTAX, is a revolutionary benchtop DNA printer that uses enzymatic technology to rapidly produce synthetic oligonucleotides, genes, pathways, and genomes. This innovative approach empowers life science researchers and labs to print their own synthetic DNA, enhancing workflow control and accelerating access to results, thereby advancing breakthroughs in life science and human health.

DNA Script SAS, established in 2014 and headquartered in Paris, France, specializes in synthetic biology tools. The company's core product, SYNTAX, is a revolutionary benchtop DNA printer that uses enzymatic technology to rapidly produce synthetic oligonucleotides, genes, pathways, and genomes. This innovative approach empowers life science researchers and labs to print their own synthetic DNA, enhancing workflow control and accelerating access to results, thereby advancing breakthroughs in life science and human health.

Deep Genomics Inc. is an artificial intelligence therapeutics company that focuses on developing individualized genetic medicines. Founded in 2014 and based in Toronto, Canada, the company utilizes advanced AI systems to enhance various stages of drug discovery and development, including target identification, lead optimization, toxicity evaluation, and trial design. Deep Genomics specializes in creating oligonucleotide therapies that address the genetic causes of diseases at the RNA and DNA levels. Its innovative AI-powered platform analyzes RNA biology to uncover new therapeutic targets and mechanisms that traditional methods cannot access, enabling the development of treatments for rare metabolic, ophthalmologic, and neurodegenerative disorders. The company recruits top talent in genomics, drug development, and AI to drive its mission of revolutionizing drug discovery.

GeneDx is a company dedicated to providing personalized and actionable health insights that enhance diagnosis and treatment, ultimately improving health outcomes. Positioned at the intersection of diagnostics and data science, GeneDx combines decades of genomic expertise with the capability to interpret clinical data at scale. The company focuses on accelerating the application of genomic information and large-scale clinical data to establish precision medicine as a standard practice in healthcare. GeneDx leads the transformation of healthcare through its comprehensive exome and genome testing services, leveraging one of the largest rare disease data sets in the world. The business operates through two segments: GeneDx, which generates the majority of its revenue, and Legacy Sema4 diagnostics.

Wugen is focused on developing off-the-shelf cellular therapies for cancer, particularly utilizing natural killer (NK) cells and CAR-T cell therapies. The company enhances NK cells obtained from healthy donors to improve their ability to target and eliminate cancer cells. Wugen's innovative approach includes a proprietary platform that leverages advanced genomic engineering to create next-generation memory NK cell therapies. These therapies specifically aim to address various types of malignancies, including solid tumors, acute myeloid leukemia, and T-cell malignancies, thereby providing new treatment options for patients facing life-threatening cancers.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

Inscripta, Inc. is a gene editing technology company based in Boulder, Colorado, with additional offices in Pleasanton and San Diego, California. Founded in 2015, Inscripta focuses on developing CRISPR enzymes, specifically MADzymes, for precision gene editing. The company has created a benchtop platform for scalable digital genome engineering, which includes an instrument, consumables, software, and assays that facilitate a fully automated workflow. This platform allows for massively parallel and trackable editing of single cells, significantly enhancing the scale and efficiency of gene editing research. By making its MAD7 CRISPR nuclease freely available for research and development, Inscripta aims to remove existing technical and licensing barriers, thereby empowering scientists and fostering a new era of biological discovery.

Omega Therapeutics is a development-stage biotechnology company based in Cambridge, Massachusetts, focused on developing genomic medicines aimed at curing diseases. The company utilizes its proprietary epigenomic programming platform to create a new class of programmable epigenetic medicines, referred to as Omega Epigenomic Controllers. These controllers are designed to selectively modulate genomic activity, enabling precise tuning of the human genome to treat various medical conditions. Founded in 2016 and rebranded from VL42, Inc. in 2017, Omega Therapeutics is dedicated to transforming human medicine by harnessing the innate potential of the genome for therapeutic benefits.

Monte Rosa Therapeutics is a biotechnology company focused on developing innovative cancer therapeutics that utilize molecular glue degraders (MGDs) to modulate protein degradation pathways. By leveraging the body's natural mechanisms for protein destruction, these small molecule drugs selectively target and degrade disease-causing proteins. The company employs its QuEEN platform, which combines artificial intelligence and proprietary experimental tools, to identify relevant proteins for degradation. With a diverse library of over 50,000 MGD molecules, Monte Rosa's lead candidate, MRT-2359, specifically targets the translation termination factor protein GSPT1, showing promise for treating MYC-driven tumors. The company aims to advance its pipeline of pioneering therapies for cancer and other diseases.

Century Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies derived from induced pluripotent stem cells (iPSCs) to treat hematologic and solid malignancies. Founded in 2018 and headquartered in Philadelphia, Pennsylvania, the company utilizes a specialized platform that integrates gene editing and protein engineering to create allogeneic therapies. This platform allows for the generation of master cell banks of modified immune effector cells, specifically engineered to optimize performance and reduce the risk of rejection by the host immune system. By employing advanced technologies such as CRISPR-mediated gene editing and proprietary chimeric antigen receptors, Century Therapeutics aims to address significant unmet medical needs in cancer and other diseases.

Gemini Therapeutics, Inc. is a precision medicine company based in Cambridge, Massachusetts, founded in 2015. The company specializes in developing new therapies for patients experiencing dry age-related macular degeneration (AMD) and related rare genetic diseases. Gemini Therapeutics focuses on genetically defined conditions, creating therapeutic candidates that address molecular abnormalities in patients with significant clinical needs. Their diverse pipeline encompasses monoclonal antibodies, recombinant proteins, and gene therapies, positioning the company to potentially introduce first-in-class therapeutics. The development of these innovative treatment options is supported by collaborations with leading life science investors and academic institutions worldwide, reflecting a commitment to advancing medical care for individuals suffering from these conditions.

Vera Therapeutics, a biotechnology company based in South San Francisco, California, specializes in developing innovative biologic therapeutics aimed at treating serious immunological diseases and genetic disorders. Founded in 2016 and renamed in March 2020, the company's research focuses on transformative treatments that can significantly improve patient outcomes. One of its key product candidates is atacicept, a fusion protein designed for self-administration via a weekly subcutaneous injection. Atacicept works by inhibiting both B lymphocyte stimulator and a proliferation-inducing ligand, which are involved in the production of autoantibodies that contribute to various autoimmune diseases. Additionally, Vera Therapeutics is exploring gene-editing technologies that hold the potential to address conditions such as sickle cell disease and cystic fibrosis.

Ikena Oncology, Inc. is a biotechnology company focused on discovering and developing biomarker-driven therapies for cancer treatment. It specializes in precision oncology by targeting pathways essential for cancer growth and therapeutic resistance, particularly within the Hippo and RAS signaling networks. The company's portfolio includes several preclinical and discovery-stage programs, such as IK-007, an EP4 receptor antagonist; IK-175, an AHR antagonist; and IK-412, a kynurenine-degrading enzyme. Additionally, Ikena is developing IK-930, an oral small-molecule inhibitor of the TEAD transcription factor involved in the Hippo pathway. Ikena Oncology, originally founded as Kyn Therapeutics in 2016, rebranded in December 2019 and is headquartered in Boston, Massachusetts. The company aims to address significant medical needs in oncology through innovative drug development.

Yumanity Therapeutics is transforming drug discovery for neurodegenerative diseases caused by protein misfolding. Founded in December 2014 by award-winning protein folding expert, Susan Lindquist, and renowned biotech industry leader, Tony Coles, Yumanity is working to identify and develop new, disease-modifying therapies that address several illnesses with critical unmet medical needs. The initial focus of the company is neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and amyotrophic lateral sclerosis (ALS). The company’s proprietary platforms have already identified one potential new target for treating Parkinson’s disease, and Yumanity is actively advancing its new chemical lead series for this condition, as well as identifying additional compounds for Alzheimer’s disease and ALS.

Taysha Gene Therapies, Inc. is a gene therapy company based in Dallas, Texas, established in 2019, focused on developing adeno-associated virus (AAV)-based gene therapies for monogenic diseases affecting the central nervous system (CNS). The company’s product pipeline includes several therapies currently in development, such as TSHA-101 for GM2 gangliosidosis, TSHA-118 for CLN1 disease, TSHA-102 for Rett syndrome, TSHA-103 for SLC6A1 haploinsufficiency disorder, and TSHA-104 for Surfeit locus 1 deficiency. Taysha Gene Therapies aims to translate innovative treatments from research to clinical practice efficiently, leveraging a strategic partnership with The University of Texas Southwestern Medical Center to enhance its development and commercialization capabilities. The company's mission is to create curative medicines that target both rare and prevalent CNS conditions, thereby addressing severe and life-threatening diseases.

Castle Creek Biosciences, Inc. is a gene therapy company headquartered in Exton, Pennsylvania, that specializes in developing and commercializing personalized gene therapies for rare skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin to create localized therapies tailored to the individual’s unique biology. This innovative approach aims to address the underlying causes of diseases that currently have high unmet medical needs, ultimately improving the lives of patients suffering from these underserved conditions.

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.

Element Biosciences, Inc., established in 2017 and headquartered in San Diego, California, specializes in developing innovative genetic analysis tools for research and diagnostic markets. The company's core business revolves around its disruptive DNA sequencing technology, which encompasses surface chemistry, sequencing chemistry, detection methods, and data analysis. Element Biosciences aims to enhance accessibility to next-generation sequencing by offering a modular, high-performing platform that delivers high-quality data and workflow flexibility, thereby reducing run and capital costs.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

10x Genomics is a life science technology company based in Pleasanton, California, that specializes in developing instruments, consumables, and software for the analysis of biological systems. Founded in 2012, the company offers a range of products, including Chromium instruments, microfluidic chips, slides, reagents, and various consumable products. Its innovative single cell solutions enable researchers to assess gene expression, immune profiling, epigenetics, and cellular heterogeneity at an individual cell level. The Visium spatial gene expression solution allows for the analysis of gene expression patterns across tissue samples, providing valuable insights into biological processes. 10x Genomics serves a diverse client base, including academic institutions, government organizations, and biopharmaceutical companies across multiple regions, including North America, Europe, and Asia. The company generates the majority of its revenue from consumables, underscoring its focus on providing essential tools for genomic research.

Castle Creek Biosciences, Inc. is a gene therapy company headquartered in Exton, Pennsylvania, that specializes in developing and commercializing personalized gene therapies for rare skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin to create localized therapies tailored to the individual’s unique biology. This innovative approach aims to address the underlying causes of diseases that currently have high unmet medical needs, ultimately improving the lives of patients suffering from these underserved conditions.

Allogene Therapeutics is a clinical-stage biotechnology company focused on developing off-the-shelf, genetically engineered allogeneic T cell therapies for cancer treatment. The company's pipeline includes UCART19, in development for relapsed/refractory acute lymphoblastic leukemia, and several preclinical allogeneic CAR T therapies targeting various cancers. Allogene's approach uses gene editing and advanced manufacturing technologies to create a scalable, broader patient eligibility option compared to autologous therapies. Revenue is primarily generated through collaborations and licensing agreements.

AgBiome, LLC is a biotechnology company based in Durham, North Carolina, that focuses on developing biological products to address significant challenges in agriculture, particularly soil-borne diseases affecting greenhouse and major row crops. Utilizing advanced genomics and screening technologies, AgBiome identifies plant-associated microbes that can enhance plant health, improve pest resistance, and increase crop yields. The company operates a state-of-the-art laboratory and greenhouse facility and collaborates with prominent agricultural firms to expedite its research and product development. AgBiome's offerings include fungicides that protect crops from various diseases and a platform for capturing and screening diverse microbes for agricultural applications. Established in 2012, AgBiome aims to provide sustainable solutions that support global food production and address critical agricultural issues such as insect and nematode control.

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.

10x Genomics is a life science technology company based in Pleasanton, California, that specializes in developing instruments, consumables, and software for the analysis of biological systems. Founded in 2012, the company offers a range of products, including Chromium instruments, microfluidic chips, slides, reagents, and various consumable products. Its innovative single cell solutions enable researchers to assess gene expression, immune profiling, epigenetics, and cellular heterogeneity at an individual cell level. The Visium spatial gene expression solution allows for the analysis of gene expression patterns across tissue samples, providing valuable insights into biological processes. 10x Genomics serves a diverse client base, including academic institutions, government organizations, and biopharmaceutical companies across multiple regions, including North America, Europe, and Asia. The company generates the majority of its revenue from consumables, underscoring its focus on providing essential tools for genomic research.

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.

Moderna, Inc. is a biotechnology company established in 2010 and headquartered in Cambridge, Massachusetts. The company specializes in developing therapeutics and vaccines using messenger RNA (mRNA) technology, which provides instructions for cells to produce proteins essential for various biological functions. Moderna has a diverse pipeline, with numerous programs targeting infectious diseases, immuno-oncology, rare diseases, and cardiovascular conditions. As of September 2024, it has 40 mRNA development candidates in clinical trials across multiple therapeutic areas. The company's mRNA technology gained significant recognition with the authorization of its COVID-19 vaccine in December 2020. Moderna maintains strategic alliances with various organizations, including major pharmaceutical companies and research institutions, to enhance its research and development efforts.

BioNTech is a Germany-based biotechnology company that specializes in developing innovative immunotherapies for cancer and other serious diseases, as well as vaccines for infectious diseases, including COVID-19. The company’s oncology pipeline includes a range of drug classes, such as mRNA-based drugs that encode antigens and neoantigens, cell therapies, bispecific antibodies, and antibody-drug conjugates. BioNTech has established partnerships with several major pharmaceutical companies, enhancing its research and development capabilities. Its COVID-19 vaccine, Comirnaty, represents its first commercialized product, demonstrating the company's commitment to addressing urgent health challenges through advanced therapeutic approaches.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Codiak BioSciences is a clinical-stage biopharmaceutical company dedicated to developing exosome-based therapeutics, a novel class of medicines with the potential to address various diseases with significant unmet medical needs. The company employs its proprietary engEx Platform, which allows for the engineering and manufacturing of exosomes, leveraging their natural role as intercellular messengers. Codiak's pipeline includes product candidates such as exoSTING and exoIL-12, targeting diverse therapeutic areas including oncology, neuro-oncology, neurology, neuromuscular diseases, and infectious diseases. Additionally, the company collaborates with the Ragon Institute to explore the potential of its exoVACC platform for vaccines against SARS-CoV-2 and HIV. Founded in 2015 and headquartered in Cambridge, Massachusetts, Codiak aims to transform therapeutic approaches by harnessing the unique properties of exosomes.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

Homology Medicines, Inc., established in 2015 and headquartered in Bedford, Massachusetts, is a biotechnology company focused on developing genetic medicines for patients with rare genetic diseases. The company's proprietary platform employs human hematopoietic stem cell-derived adeno-associated virus vectors (AAVHSCs) to deliver genetic therapies or nuclease-free gene editing treatments in vivo, targeting various disease-relevant tissues such as the liver, central nervous system, and eye. Homology Medicines has two lead product candidates: HMI-102, currently in a Phase 1/2 clinical trial for treating phenylketonuria (PKU) in adults, and HMI-103, intended for PKU treatment in pediatric patients. Additionally, the company is developing HMI-202 to treat metachromatic leukodystrophy. Homology Medicines aims to cure the underlying causes of these diseases by leveraging its robust intellectual property portfolio and experienced management team with a proven track record in rare disease therapeutics.

Translate Bio, Inc. is a clinical-stage biotechnology company focused on developing messenger RNA (mRNA) therapeutics aimed at addressing diseases linked to protein or gene dysfunction. Founded in 2011 and headquartered in Lexington, Massachusetts, the company is advancing its lead candidates, MRT5005 and MRT5201. MRT5005 is currently undergoing Phase I/II clinical trials for the treatment of cystic fibrosis, while MRT5201 is being developed for ornithine transcarbamylase deficiency. Translate Bio was previously known as RaNA Therapeutics, Inc. before rebranding in June 2017. The company specializes in innovative therapies for rare diseases, leveraging the potential of RNA-based medicine.

Castle Creek Biosciences, Inc. is a gene therapy company headquartered in Exton, Pennsylvania, that specializes in developing and commercializing personalized gene therapies for rare skin and connective tissue disorders. Founded in 2015, the company utilizes a fibroblast technology platform that extracts fibroblast cells from a patient's own skin to create localized therapies tailored to the individual’s unique biology. This innovative approach aims to address the underlying causes of diseases that currently have high unmet medical needs, ultimately improving the lives of patients suffering from these underserved conditions.

10x Genomics is a life science technology company based in Pleasanton, California, that specializes in developing instruments, consumables, and software for the analysis of biological systems. Founded in 2012, the company offers a range of products, including Chromium instruments, microfluidic chips, slides, reagents, and various consumable products. Its innovative single cell solutions enable researchers to assess gene expression, immune profiling, epigenetics, and cellular heterogeneity at an individual cell level. The Visium spatial gene expression solution allows for the analysis of gene expression patterns across tissue samples, providing valuable insights into biological processes. 10x Genomics serves a diverse client base, including academic institutions, government organizations, and biopharmaceutical companies across multiple regions, including North America, Europe, and Asia. The company generates the majority of its revenue from consumables, underscoring its focus on providing essential tools for genomic research.

Codiak BioSciences is a clinical-stage biopharmaceutical company dedicated to developing exosome-based therapeutics, a novel class of medicines with the potential to address various diseases with significant unmet medical needs. The company employs its proprietary engEx Platform, which allows for the engineering and manufacturing of exosomes, leveraging their natural role as intercellular messengers. Codiak's pipeline includes product candidates such as exoSTING and exoIL-12, targeting diverse therapeutic areas including oncology, neuro-oncology, neurology, neuromuscular diseases, and infectious diseases. Additionally, the company collaborates with the Ragon Institute to explore the potential of its exoVACC platform for vaccines against SARS-CoV-2 and HIV. Founded in 2015 and headquartered in Cambridge, Massachusetts, Codiak aims to transform therapeutic approaches by harnessing the unique properties of exosomes.

Syros Pharmaceuticals, Inc. is a biopharmaceutical company based in Cambridge, Massachusetts, that specializes in developing innovative treatments for cancer and monogenic diseases through gene control medicines. The company is advancing its lead product candidates, including SY-1425, a selective retinoic acid receptor alpha agonist currently in Phase II clinical trials for specific patient populations with acute myeloid leukemia and myelodysplastic syndrome, and SY-5609, a cyclin-dependent kinase 7 inhibitor under investigation in a Phase I clinical trial for advanced solid tumors. Syros is also engaged in collaborative research, including a partnership with Incyte Corporation aimed at identifying therapeutic targets for myeloproliferative neoplasms and a licensing agreement with TMRC Co. Ltd. for the development of tamibarotene. With a focus on mapping gene regulatory circuits, Syros leverages its proprietary platform and expertise to address significant unmet medical needs in oncology. Founded in 2011, the company aims to establish new standards of care for patients with hematologic malignancies.

Allena Pharmaceuticals is a late-stage clinical biopharmaceutical company based in Newton, Massachusetts, that specializes in developing and commercializing oral enzyme therapeutics for rare and severe metabolic and kidney disorders. The company's primary focus is on conditions that involve the excessive accumulation of metabolites such as oxalate and urate, which can lead to complications like kidney stones and chronic kidney disease. Allena's lead product candidate, reloxaliase, is currently undergoing a pivotal Phase 3 clinical program aimed at treating enteric hyperoxaluria, a disorder linked to significantly high urinary oxalate levels. Additionally, the company is developing ALLN-346, a novel enzyme designed to address hyperuricemia in patients with moderate to severe chronic kidney diseases. Allena Pharmaceuticals aims to provide innovative treatments that address unmet medical needs in these patient populations.

Codiak BioSciences is a clinical-stage biopharmaceutical company dedicated to developing exosome-based therapeutics, a novel class of medicines with the potential to address various diseases with significant unmet medical needs. The company employs its proprietary engEx Platform, which allows for the engineering and manufacturing of exosomes, leveraging their natural role as intercellular messengers. Codiak's pipeline includes product candidates such as exoSTING and exoIL-12, targeting diverse therapeutic areas including oncology, neuro-oncology, neurology, neuromuscular diseases, and infectious diseases. Additionally, the company collaborates with the Ragon Institute to explore the potential of its exoVACC platform for vaccines against SARS-CoV-2 and HIV. Founded in 2015 and headquartered in Cambridge, Massachusetts, Codiak aims to transform therapeutic approaches by harnessing the unique properties of exosomes.

23andMe, Inc. is a consumer genetics and research company headquartered in Sunnyvale, California, founded in 2006. It specializes in providing direct-to-consumer personal genome services through home-based saliva collection kits. The company offers a range of services, including Health + Ancestry, which provides insights into ancestry, traits, and health, and Ancestry + Traits, which details ancestry breakdown and various trait reports. 23andMe aims to help individuals understand their genetic makeup, enabling users to explore their ancestry, genealogy, and inherited traits. Additionally, the company markets its services to researchers and scientists, offering categorized and searchable genetic data. With a focus on consumer and research services, 23andMe is positioned within the rapidly growing biotech and healthcare sectors, contributing to the understanding of genetics and its implications for health.

Intellia Therapeutics, Inc. is a genome editing company headquartered in Cambridge, Massachusetts, specializing in the development of therapeutics using the CRISPR/Cas9 technology. This innovative platform allows for precise alterations of genomic DNA, enabling the company to pursue treatments for a range of severe and life-threatening diseases. Intellia's pipeline includes both in vivo programs targeting liver diseases such as transthyretin amyloidosis and hereditary angioedema, as well as ex vivo programs focused on engineered cell therapies for oncological and autoimmune disorders. The company is also exploring treatments for conditions like primary hyperoxaluria Type 1, alpha-1 antitrypsin deficiency, and Hemophilia B. Intellia has established collaborations with several organizations, enhancing its research and development capabilities. Founded in 2014, Intellia holds a significant intellectual property portfolio related to the therapeutic applications of CRISPR/Cas9, aiming to address high unmet medical needs through its advanced gene-editing approaches.

Wave Life Sciences Ltd. is a clinical-stage genetic medicine company focused on developing innovative RNA-based therapies. Utilizing its proprietary PRISM platform, the company designs, optimizes, and produces stereopure oligonucleotides aimed at addressing genetic defects. By targeting ribonucleic acid (RNA), Wave aims to either diminish the production of harmful proteins or convert dysfunctional proteins into functional ones. The company's primary focus is on neurology, particularly disorders affecting the central and neuromuscular systems. Wave Life Sciences has established research and collaboration agreements with major pharmaceutical companies, including Pfizer and Takeda, to further advance its therapeutic programs. Founded in 2012 and headquartered in Singapore, Wave Life Sciences is dedicated to delivering transformative treatments for patients with genetically defined diseases.

Ovid Therapeutics Inc. is a biopharmaceutical company based in New York, focused on developing innovative therapies for patients with neurological disorders, particularly rare diseases. Founded in 2014, the company is advancing a promising pipeline of drug candidates aimed at treating conditions such as angelman syndrome and fragile X syndrome. Its lead candidate, OV101, is in Phase III clinical trials for angelman syndrome and has completed Phase II trials for fragile X syndrome. Additionally, Ovid is developing OV935, currently in Phase II trials for cyclin-dependent kinase-like 5 deficiency disorder and Dravet syndrome. The company is also researching OV329 for infantile spasms and OV881 as a microRNA gene therapy for angelman syndrome. Ovid Therapeutics has established collaborations and licensing agreements with various pharmaceutical and research organizations to enhance its development efforts. The company's commitment to addressing unmet medical needs is underpinned by strong intellectual property and a focus on safe, effective treatments for patients facing challenging neurological conditions.

Editas Medicine is a clinical-stage biotechnology company based in Cambridge, Massachusetts, with an additional site in Boulder, Colorado. Founded in 2013, the company focuses on the discovery and development of genome editing therapeutics, primarily utilizing its proprietary CRISPR/Cas9 technology. This innovative platform enables precise molecular modifications at the genetic level, with the aim of treating a wide array of serious diseases by addressing their underlying causes. Editas Medicine has established a strong intellectual property portfolio, which includes numerous patents related to foundational genome editing technologies and advancements that facilitate the translation of research into viable human therapeutics. The company's mission is to pioneer transformative genomic medicines that can significantly improve patient outcomes across various genetic disorders.

GenSight Biologics S.A. is a clinical-stage biotechnology company based in Paris, France, focused on developing gene therapy treatments for retinal degenerative diseases and neurodegenerative conditions affecting the central nervous system. Utilizing its core technology platforms—Mitochondrial Targeting Sequence and Optogenetics—the company aims to preserve and restore vision in patients with severe retinal disorders. Its lead product candidates include GS010, an AAV2-based gene therapy currently in Phase III trials for treating Leber hereditary optic neuropathy caused by mutations in the ND4 gene, and GS030, which is undergoing Phase I/II trials for retinitis pigmentosa and geographic atrophy in dry age-related macular degeneration. In addition to these, GenSight is advancing several products in preclinical stages targeting various ophthalmic and neurodegenerative diseases. Founded in 2012, the company is dedicated to innovative approaches that aim to prevent vision loss and improve the quality of life for affected patients.

Kura Oncology is a clinical-stage biopharmaceutical company based in San Diego, California, focused on developing targeted therapies for cancer treatment. The company’s primary product candidate, Tipifarnib, is an orally available farnesyl transferase inhibitor currently undergoing Phase II clinical trials for solid tumors, peripheral T-cell lymphomas, and various hematologic malignancies. In addition to Tipifarnib, Kura is advancing other small molecule candidates, including KO-947, which targets dysregulated extracellular signal-related kinases, and KO-539, aimed at inhibiting the menin-mixed lineage leukemia protein interaction. Kura Oncology emphasizes precision medicine, leveraging insights from cancer genomics to create targeted treatments for patients with specific genetic profiles, addressing significant unmet medical needs across various cancer types, including lung, colorectal, thyroid, pancreatic cancers, and blood cancers such as lymphoma and leukemia. The company was founded in 2014 and operates primarily within the United States.