Eli Lilly and Company

Rona Therapeutics is an innovative RNA therapeutics platform focused on the discovery, development, and commercialization of modular and programmable RNA medicines. The company specializes in oligonucleotide therapies, RNA editing, and polymeric molecules, aiming to create transformative RNA therapies that can significantly impact disease treatment across various therapeutic areas. Rona Therapeutics is committed to developing proprietary technologies that enhance the specificity, stability, and longevity of therapeutic effects. To support its mission, the company is establishing state-of-the-art facilities to advance nucleic acid synthesis, delivery platforms, and RNA biology. Through its efforts, Rona Therapeutics seeks to improve patient health outcomes and foster an environment conducive to innovation and excellence in RNA medicine.

Founded in 2020 and headquartered in Boston, Massachusetts, Scorpion Therapeutics is a clinical-stage biotechnology company specializing in precision oncology. It develops targeted small-molecule drugs to treat cancer by focusing on three areas: therapies against known oncogenes, agents for currently undruggable targets, and drugs for new targets.

Avenzo Therapeutics focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

ProQR Therapeutics is a biopharmaceutical company focused on developing RNA-based therapies for patients with severe genetic rare diseases. Its primary focus includes Leber's congenital amaurosis type 10, dystrophic epidermolysis bullosa, and other inherited retinal disorders.

HAYA Therapeutics develops RNA-based therapeutics focused on treating fibrotic diseases. Its precision medicines target specific regulators of fibrosis in tissues like the heart, aiming to block fibrosis progression and treat associated conditions safely and effectively.

Positive Sequence Biology is a biotech company with new gene system-based editing, focused on biotech innovation & breakthrough therapies.

Rona Therapeutics is an innovative RNA therapeutics platform focused on the discovery, development, and commercialization of modular and programmable RNA medicines. The company specializes in oligonucleotide therapies, RNA editing, and polymeric molecules, aiming to create transformative RNA therapies that can significantly impact disease treatment across various therapeutic areas. Rona Therapeutics is committed to developing proprietary technologies that enhance the specificity, stability, and longevity of therapeutic effects. To support its mission, the company is establishing state-of-the-art facilities to advance nucleic acid synthesis, delivery platforms, and RNA biology. Through its efforts, Rona Therapeutics seeks to improve patient health outcomes and foster an environment conducive to innovation and excellence in RNA medicine.

Radar Therapeutics is a pioneering company specializing in precision-expressed mRNA-based therapeutics designed to combat pathogenic cells by targeting their unique transcriptional signatures. The company has developed advanced ribonucleic acid sensing technology, which incorporates control elements for creating rationally designed, precise, and programmable therapies. This innovative approach allows for the targeting of previously undruggable therapeutic targets, enabling healthcare professionals to administer treatments that can either eliminate or reprogram specific pathogenic cells effectively. Through its groundbreaking advancements, Radar Therapeutics aims to provide novel solutions for complex diseases, fundamentally changing the landscape of therapeutic development.

IMPACT Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics for cancer and other life-threatening diseases. Founded in 2009 and based in Shanghai, China, the company focuses on creating targeted anti-cancer treatments utilizing synthetic lethality. IMPACT Therapeutics is actively engaged in several research and development projects, including a PARP inhibitor, a Wee1 inhibitor, and various novel agents targeting DNA damage response mechanisms and the Hedgehog signaling pathway. Through its commitment to advancing treatment options, IMPACT Therapeutics aims to provide patients with effective and targeted anti-cancer therapies.

Beijing Targeting One Technology Co., Ltd. specializes in the development and production of biomedical instruments and consumables, focusing on in vitro diagnostic reagents for precision medicine, health management, and life science research. Established in 2015 and based in Beijing, the company offers a range of products including sample preparation apparatus, biochip readers, digital PCR supplies, and genetic testing kits. Its digital PCR solutions play a crucial role in medical diagnostics, with applications in transplant rejection monitoring, targeted sequencing, tumor liquid biopsy, and non-invasive prenatal screening. Targeting One aims to enhance gene testing and precision medicine through its innovative technologies and comprehensive offerings.

Laverock Therapeutics develops a gene silencing platform for creating programmable, allogeneic cell therapies.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

Amber Bio is a biotechnology company pioneering new gene editing modalities using multi-kilobase edits to reach previously undruggable patient populations.

Sigilon Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing functional cures for patients with chronic diseases. Founded in 2015, the company aims to create immune-protected, engineered human cells that restore normal physiological functions without the risk of fibrosis or immune rejection. Its lead product candidate, SIG-001, is currently in Phase I/II clinical trials for preventing bleeding episodes in patients with moderate to severe Hemophilia A. Additionally, Sigilon is advancing other candidates, including SIG-005, which targets non-neurological manifestations of mucopolysaccharidosis type 1, and SIG-002, designed to replace islet cells for treating type 1 diabetes. The company's innovative Shielded Living Therapeutics platform leverages advances in cell engineering and proprietary biocompatible materials to offer stable and durable therapeutic solutions for a variety of chronic conditions.

Verve Therapeutics is a biotechnology company developing genetic medicines for cardiovascular disease, aiming to transform treatment from chronic management to single-course gene editing therapies. It leverages human genetics analysis and gene-editing technology, with expertise in cardiovascular medicine, human genetics, gene editing, delivery technologies, drug development, and commercialization.

Avenzo Therapeutics focuses on developing innovative cancer treatments. It operates a clinical-stage platform dedicated to creating novel therapies for underserved cancers, with a pipeline targeting rare and genomically defined cancers.

Switch Therapeutics is a biotechnology company founded in 2020 and based in San Francisco, California. The company focuses on revolutionizing RNA interference (RNAi) therapies by developing biomarker-gated genetic medicines. These innovative therapies utilize nucleic acid nanotechnology and RNAi science to target a variety of diseases, particularly those affecting the central nervous system and other systemic conditions with significant unmet medical needs. Switch Therapeutics aims to provide healthcare providers with RNA molecules and therapies that can be activated selectively in specific cells, enhancing the precision of treatments for central nervous system diseases.

Volastra Therapeutics, Inc. is a biotechnology company focused on developing innovative therapies for metastatic cancers. Established in 2019 and headquartered in New York, the company leverages unique insights into chromosomal instability to create targeted treatment strategies. Volastra utilizes a library of organoids derived from metastatic cancer samples to better understand tumor spread and identify effective therapeutic approaches. Additionally, the company combines artificial intelligence, bioinformatics, and proprietary imaging techniques, which enhance the ability of healthcare professionals to determine which cancers are more likely to metastasize. This approach aims to facilitate quicker target identification, optimize patient selection for therapies, and ultimately improve patient outcomes.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

ProQR Therapeutics is a biopharmaceutical company focused on developing RNA-based therapies for patients with severe genetic rare diseases. Its primary focus includes Leber's congenital amaurosis type 10, dystrophic epidermolysis bullosa, and other inherited retinal disorders.

SonoThera is a biotechnology company focused on developing innovative genetic therapies using ultrasound technology. The company's mission is to create non-viral genetic medicines that address the root causes of human diseases. SonoThera specializes in ultrasound-guided nonviral gene therapy, aiming to provide effective treatments for conditions that currently have suboptimal or no available treatments. By leveraging this approach, SonoThera seeks to enhance the health and quality of life for millions of people worldwide.

Rgenta Therapeutics, Inc. is a biotechnology company focused on developing RNA-targeting medicines aimed at treating oncology and neurological disorders. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes a proprietary platform that analyzes extensive genomic data to identify targetable RNA processing events. This innovative approach enables the design of small-molecule glues that modulate interactions among the spliceosome, regulatory proteins, and RNAs. Rgenta's unique methodology aims to unlock the therapeutic potential of previously undruggable targets, ultimately advancing drug development for human diseases.

Strand Therapeutics Inc. is a biotechnology company based in Boston, Massachusetts, that specializes in genetically programming mRNA to deliver innovative therapies aimed at enhancing patient outcomes. Founded in 2017, the company develops mRNA programming technology that allows for precise control over the location, timing, and intensity of therapeutic protein expression within the body. By employing mRNA-encoded logic circuits, Strand Therapeutics can achieve cell-type specific expression by detecting unique miRNA signatures and modulating protein dosage in response to externally administered small molecules. The company focuses on creating gene therapies powered by synthetic biology, including immunotherapies designed to enable cells to produce target cancer-killing proteins, thereby strengthening the body's immune response against cancer.

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.

Akouos is a biotechnology company focused on precision genetic medicine to restore and preserve hearing. Based in Boston, it develops gene therapies using a proprietary adeno-associated viral vector platform and delivery approaches to treat sensorineural hearing loss in genetically defined populations. The company's lead program, AK-OTOF, targets hearing loss caused by mutations in the OTOF gene. Akouos pursues research and development across gene-based interventions aimed at improving auditory function and long-term sensory outcomes. It has collaborations with academic and industry partners, including Massachusetts Eye and Ear and Lonza, to advance discovery and manufacturing capabilities. Founded in 2016, Akouos addresses unmet clinical needs in auditory health by seeking to restore physiologic hearing through targeted genetic therapies.

Rona Therapeutics is an innovative RNA therapeutics platform focused on the discovery, development, and commercialization of modular and programmable RNA medicines. The company specializes in oligonucleotide therapies, RNA editing, and polymeric molecules, aiming to create transformative RNA therapies that can significantly impact disease treatment across various therapeutic areas. Rona Therapeutics is committed to developing proprietary technologies that enhance the specificity, stability, and longevity of therapeutic effects. To support its mission, the company is establishing state-of-the-art facilities to advance nucleic acid synthesis, delivery platforms, and RNA biology. Through its efforts, Rona Therapeutics seeks to improve patient health outcomes and foster an environment conducive to innovation and excellence in RNA medicine.

IMPACT Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics for cancer and other life-threatening diseases. Founded in 2009 and based in Shanghai, China, the company focuses on creating targeted anti-cancer treatments utilizing synthetic lethality. IMPACT Therapeutics is actively engaged in several research and development projects, including a PARP inhibitor, a Wee1 inhibitor, and various novel agents targeting DNA damage response mechanisms and the Hedgehog signaling pathway. Through its commitment to advancing treatment options, IMPACT Therapeutics aims to provide patients with effective and targeted anti-cancer therapies.

Arkuda Therapeutics, Inc. is a biotechnology company founded in 2018 and based in Cambridge, Massachusetts, focused on developing innovative treatments for patients with neurological diseases. The company utilizes new insights into progranulin and lysosomal biology to create therapies that aim to correct progranulin deficiency and address lysosomal dysfunction, particularly in genetically-defined frontotemporal dementia associated with mutations in the GRN gene. By targeting these underlying biological mechanisms, Arkuda's lead compounds are designed to improve cellular health in the brain, potentially delaying the progression of neurodegenerative diseases and the onset of their symptoms.

Verge Genomics is a biotechnology company that uses artificial intelligence and machine learning to accelerate drug discovery for neurodegenerative diseases. The company is founded by experts in machine learning and neuroscience drug development who believe advances in computational genomics can reveal new targets and therapies. Verge Genomics' platform analyzes datasets of any size to identify targets and predict effective drugs, with the aim of delivering treatments that improve patient outcomes while reducing the cost and duration of pharmaceutical development.

Singleron Biotechnologies Ltd. is a molecular diagnostic company specializing in single cell sequencing solutions. Founded in 2018 and based in Nanjing, China, with additional offices and laboratories in Suzhou, the United States, and Germany, the firm develops a range of innovative products, including the Singleron Matrix automated instrument for high-density microwell processing and GEXSCOPE single cell RNA sequencing kits. The company also offers a microfluidic chip for cell partitioning, magnetic beads for cell barcoding, and tools for data analysis such as CeleScope and SynEcoSys, which facilitate the interpretation of single cell sequencing data. Singleron provides services in single cell RNA sequencing, single nucleus RNA sequencing, and bioinformatics analysis, aiming to advance clinical diagnosis, drug development, and health management. The company collaborates with research institutes, hospitals, and pharmaceutical firms primarily in China and the United States, leveraging its exclusive IP license from Yale University to enhance its innovative capabilities in single cell analysis.

Positive Sequence Biology is a biotech company with new gene system-based editing, focused on biotech innovation & breakthrough therapies.

Hopstem Biotechnology LLC is a Houston-based company that focuses on the development of neural stem cell technology and stem cell therapies specifically for neurological disorders. Established in 2019, the company specializes in the neural differentiation, cell banking, and cell engineering of human induced pluripotent stem cells (iPSCs) and human embryonic stem cells (ESCs). By leveraging its advanced technologies, Hopstem aims to enhance biomedical research and improve diagnostic and therapeutic options for various neurological conditions. Through its innovative approaches, the company seeks to provide effective treatment solutions for patients suffering from these disorders.

GenEdit Inc., established in 2016 and headquartered in Berkeley, California, specializes in developing innovative genome editing tools. The company's core technology is CRISPR/Cas9, which enables precise therapeutic gene editing. GenEdit has further advanced this field by creating the NanoGalaxy™ platform, a proprietary non-viral delivery system that efficiently transports CRISPR/Cas9 to target tissues, overcoming previous delivery challenges. This platform systematically screens a library of nanoparticles to ensure safer and more effective delivery, making therapeutic gene editing easier, faster, and more accurate than traditional methods. GenEdit's ultimate goal is to facilitate the cure of previously incurable genetic diseases by providing physicians with powerful tools for gene therapy.

Vanqua Bio is a biotechnology company dedicated to discovering and developing innovative medicines for patients with neurodegenerative diseases. The company's proprietary platform employs human genetics and patient-derived neuronal cells to identify and validate novel disease pathways related to lysosomal dysfunction or aberrant immune system activation, aiming to translate these findings into clinically effective treatments.

ADARx Pharmaceuticals specializes in the development of RNA editing therapeutics. Founded in 2019, the company focuses on discovering and developing innovative treatments that utilize endogenous enzymes to precisely target and correct single point mutations in mRNA transcripts, restoring the production of functional proteins.

Abogen Biosciences, established in 2019 and headquartered in Jiangsu, China, specializes in the development of nucleic acid-based therapeutics, including RNA and DNA treatments, for combating cancer and infectious diseases.

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.

MiNA Therapeutics Limited is a clinical-stage biotechnology company based in London, United Kingdom, focused on the design and development of small activating RNA medicines aimed at restoring normal cellular function. Founded in 2008, the company is dedicated to harnessing gene activation mechanisms to create therapeutic solutions for cancer and other severe diseases. Its product pipeline includes MTL-CEBPA combined with Sorafenib for hepatocellular carcinoma and MTL-CEBPA paired with Pembrolizumab for advanced solid tumors. Through its innovative approach, MiNA Therapeutics aims to transform the treatment landscape, enabling healthcare professionals to effectively address challenging medical conditions.

Rgenta Therapeutics, Inc. is a biotechnology company focused on developing RNA-targeting medicines aimed at treating oncology and neurological disorders. Founded in 2018 and based in Cambridge, Massachusetts, the company utilizes a proprietary platform that analyzes extensive genomic data to identify targetable RNA processing events. This innovative approach enables the design of small-molecule glues that modulate interactions among the spliceosome, regulatory proteins, and RNAs. Rgenta's unique methodology aims to unlock the therapeutic potential of previously undruggable targets, ultimately advancing drug development for human diseases.

EdiGene Inc. is a biotechnology company specializing in the development of genome editing technologies for therapeutic applications in cancer and various genetic diseases. Founded in 2015 and headquartered in Beijing, China, with additional offices in Guangzhou and Cambridge, Massachusetts, EdiGene focuses on both ex vivo and in vivo gene-editing therapies. The company has established proprietary platforms for editing hematopoietic stem cells and T cells, as well as an RNA base editing approach for in vivo applications. EdiGene's innovative methods enable targeted modifications of genetic information in human cells and facilitate high-throughput drug discovery through advanced screening techniques. By translating these technologies into novel therapeutics, EdiGene aims to improve the safety and efficacy of gene therapy, ultimately benefiting patients, the pharmaceutical industry, and the scientific research community.

Jaguar Gene Therapy is developing gene therapy treatments for severe genetic diseases. Its clinical-stage pipeline focuses on neurodevelopmental disorders, Type 1 galactosemia, and Type 1 diabetes.

Qihan Biotech is a biomedical company specializing in genetic technology with a focus on addressing the global shortage of organ donors. The company aims to produce safe and effective cells, tissues, and organs for human transplantation using advanced gene editing techniques. By applying these technologies to xenogenic organ transplantation, Qihan Biotech seeks to create organs that can be transplanted into humans, thereby reducing the need for traditional organ transplants and saving lives.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

Evox Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, that specializes in developing exosome-based therapeutics for the treatment of severe diseases with limited treatment options. Founded in 2016, the company focuses on harnessing and engineering extracellular vesicles, known as exosomes, to facilitate targeted delivery of nucleic acids and proteins. Evox's innovative platform allows for the modification of exosomes using advanced molecular engineering techniques, enabling effective drug delivery to specific organs, including the brain and central nervous system. By leveraging these natural delivery capabilities, Evox aims to overcome the limitations associated with conventional protein, antibody, and nucleic acid therapies, thereby creating novel therapeutic solutions that could significantly impact human health. The company is supported by a robust intellectual property portfolio and is positioned as a leader in this emerging therapeutic space.

Seraxis is a private biotechnology company focused on cell replacement therapies for insulin-dependent diabetes. Founded in 2013, it operates across Singapore and the United States, with a GMP laboratory in Germantown, Maryland. The company develops novel cell and encapsulation technologies to enable a practical cell therapy that could provide long-term diabetes control without the need for immunosuppression. Its work centers on producing insulin through engineered cells to regulate glucose levels, aiming for a durable cure that reduces or eliminates ongoing immunosuppressive treatment. Seraxis emphasizes advancing development quickly while pursuing value creation through its structure and activities.

Beijing Targeting One Technology Co., Ltd. specializes in the development and production of biomedical instruments and consumables, focusing on in vitro diagnostic reagents for precision medicine, health management, and life science research. Established in 2015 and based in Beijing, the company offers a range of products including sample preparation apparatus, biochip readers, digital PCR supplies, and genetic testing kits. Its digital PCR solutions play a crucial role in medical diagnostics, with applications in transplant rejection monitoring, targeted sequencing, tumor liquid biopsy, and non-invasive prenatal screening. Targeting One aims to enhance gene testing and precision medicine through its innovative technologies and comprehensive offerings.

SciNeuro Pharmaceuticals is a biotechnology company based in Shanghai, China, focused on developing innovative medicines for patients with central nervous system (CNS) diseases. The company is committed to transforming the treatment landscape for these conditions through a robust product pipeline that emphasizes internal innovation and global collaboration. SciNeuro's drug development is guided by breakthrough scientific evidence, including insights from human genetics and pharmacology, targeting the underlying mechanisms of CNS diseases. By addressing these pathological mechanisms, SciNeuro aims to provide transformative therapies that not only aim to cure these diseases but also enhance the quality of life for patients.

IMPACT Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics for cancer and other life-threatening diseases. Founded in 2009 and based in Shanghai, China, the company focuses on creating targeted anti-cancer treatments utilizing synthetic lethality. IMPACT Therapeutics is actively engaged in several research and development projects, including a PARP inhibitor, a Wee1 inhibitor, and various novel agents targeting DNA damage response mechanisms and the Hedgehog signaling pathway. Through its commitment to advancing treatment options, IMPACT Therapeutics aims to provide patients with effective and targeted anti-cancer therapies.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

EdiGene Inc. is a biotechnology company specializing in the development of genome editing technologies for therapeutic applications in cancer and various genetic diseases. Founded in 2015 and headquartered in Beijing, China, with additional offices in Guangzhou and Cambridge, Massachusetts, EdiGene focuses on both ex vivo and in vivo gene-editing therapies. The company has established proprietary platforms for editing hematopoietic stem cells and T cells, as well as an RNA base editing approach for in vivo applications. EdiGene's innovative methods enable targeted modifications of genetic information in human cells and facilitate high-throughput drug discovery through advanced screening techniques. By translating these technologies into novel therapeutics, EdiGene aims to improve the safety and efficacy of gene therapy, ultimately benefiting patients, the pharmaceutical industry, and the scientific research community.

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.

Nido Biosciences is a clinical-stage biopharmaceutical company based in Boston, Massachusetts, focused on developing precision medicines and small-molecule therapeutics for severe neurological and neuromuscular diseases. It employs a functional genomics platform using human cell lines and tailored screens to identify therapeutic targets driven by human genetics, with the aim of addressing the underlying biology of disease and restoring healthy cell function through targeted interventions.

Singleron Biotechnologies Ltd. is a molecular diagnostic company specializing in single cell sequencing solutions. Founded in 2018 and based in Nanjing, China, with additional offices and laboratories in Suzhou, the United States, and Germany, the firm develops a range of innovative products, including the Singleron Matrix automated instrument for high-density microwell processing and GEXSCOPE single cell RNA sequencing kits. The company also offers a microfluidic chip for cell partitioning, magnetic beads for cell barcoding, and tools for data analysis such as CeleScope and SynEcoSys, which facilitate the interpretation of single cell sequencing data. Singleron provides services in single cell RNA sequencing, single nucleus RNA sequencing, and bioinformatics analysis, aiming to advance clinical diagnosis, drug development, and health management. The company collaborates with research institutes, hospitals, and pharmaceutical firms primarily in China and the United States, leveraging its exclusive IP license from Yale University to enhance its innovative capabilities in single cell analysis.

Evox Therapeutics Limited is a biotechnology company based in Oxford, United Kingdom, that specializes in developing exosome-based therapeutics for the treatment of severe diseases with limited treatment options. Founded in 2016, the company focuses on harnessing and engineering extracellular vesicles, known as exosomes, to facilitate targeted delivery of nucleic acids and proteins. Evox's innovative platform allows for the modification of exosomes using advanced molecular engineering techniques, enabling effective drug delivery to specific organs, including the brain and central nervous system. By leveraging these natural delivery capabilities, Evox aims to overcome the limitations associated with conventional protein, antibody, and nucleic acid therapies, thereby creating novel therapeutic solutions that could significantly impact human health. The company is supported by a robust intellectual property portfolio and is positioned as a leader in this emerging therapeutic space.

Legend Biotech is a clinical-stage biopharmaceutical company focused on discovering and developing novel cell therapies for oncology and other indications. Its lead candidate, LCAR-B38M/JNJ-4528, is an autologous CAR-T therapy for multiple myeloma, evaluated in various trials including earlier-line settings and comparisons with standard therapies in Revlimid-refractory disease. The company also has a broad pipeline of autologous and allogeneic CAR-T and related immunotherapies targeting solid tumors and hematologic cancers, including investigations into CD20-directed allogeneic CAR-T for non-Hodgkin lymphoma in China as an investigator-initiated Phase 1 study, as well as early preclinical and clinical programs for additional cancers and infectious diseases. Legend Biotech was founded in 2014 and is based in Somerset, New Jersey; it is a subsidiary of Genscript Biotech Corporation.

Sigilon Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing functional cures for patients with chronic diseases. Founded in 2015, the company aims to create immune-protected, engineered human cells that restore normal physiological functions without the risk of fibrosis or immune rejection. Its lead product candidate, SIG-001, is currently in Phase I/II clinical trials for preventing bleeding episodes in patients with moderate to severe Hemophilia A. Additionally, Sigilon is advancing other candidates, including SIG-005, which targets non-neurological manifestations of mucopolysaccharidosis type 1, and SIG-002, designed to replace islet cells for treating type 1 diabetes. The company's innovative Shielded Living Therapeutics platform leverages advances in cell engineering and proprietary biocompatible materials to offer stable and durable therapeutic solutions for a variety of chronic conditions.

Immunocore is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

DTx Pharma, LLC is a biotechnology company headquartered in San Diego, California, specializing in the development and commercialization of fatty acid-conjugated peptide therapeutics. Founded in 2017, the company focuses on delivering RNA-based medicines to treat patients with rare and chronic diseases, including retinitis pigmentosa, Duchenne muscular dystrophy, and various central nervous system disorders. DTx Pharma's innovative delivery technology addresses challenges faced by previous-generation platforms, such as poor pharmacokinetics and limited cellular uptake, thereby enhancing the efficiency of nucleic acid drug delivery across diverse tissues and cell types. The company aims to establish RNA therapeutics as a leading option for personalized treatment in multiple therapeutic areas.

BGI is a biotechnology company specializing in genomics. It provides genome sequencing and genetic testing services to research institutions and healthcare professionals worldwide. The company's offerings include bioinformatics, multi-omics solutions, and related tools, catering to a diverse range of clients such as pharmaceutical companies and academic institutions. BGI's work has contributed significantly to genetic and genomic research, with notable collaborations including Eli Lilly and Company, Merck, Pfizer Inc., and the Bill & Melinda Gates Foundation.

Geneseeq Technology Inc., founded in 2008 and based in Toronto, Canada, is a clinical testing and diagnostic center specializing in next-generation sequencing (NGS) technologies for precision medicine in cancer treatment. The company's platform translates patients' genomic information into decision-making tools for physicians and cancer patients. Geneseeq's laboratories have achieved numerous qualifications, including passing assessments from the China’s National Center of Clinical Laboratories, College of American Pathologists, and European Molecular Genetics Quality Network.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

EdiGene Inc. is a biotechnology company specializing in the development of genome editing technologies for therapeutic applications in cancer and various genetic diseases. Founded in 2015 and headquartered in Beijing, China, with additional offices in Guangzhou and Cambridge, Massachusetts, EdiGene focuses on both ex vivo and in vivo gene-editing therapies. The company has established proprietary platforms for editing hematopoietic stem cells and T cells, as well as an RNA base editing approach for in vivo applications. EdiGene's innovative methods enable targeted modifications of genetic information in human cells and facilitate high-throughput drug discovery through advanced screening techniques. By translating these technologies into novel therapeutics, EdiGene aims to improve the safety and efficacy of gene therapy, ultimately benefiting patients, the pharmaceutical industry, and the scientific research community.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.

Avidity Biosciences is a biopharmaceutical company developing antibody oligonucleotide conjugates to enable tissue-selective delivery and precision RNA therapies for serious diseases. Its AOC platform combines monoclonal antibody tissue targeting with oligonucleotide therapies to access previously undruggable tissues and genetic drivers. The lead candidate, AOC 1001, targets myotonic dystrophy type 1, and its muscle programs address muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. The pipeline also includes immune and other cell-type programs, including Del-zota for DMD, Del-desiran for DM1, and Del-brax for FSHD. Avidity is based in La Jolla, California.

Gracell Biotechnologies Inc. is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative cell therapies for cancer treatment. Based in Shanghai, China, Gracell's lead product candidates include GC012F, a dual-targeting autologous CAR-T therapy for multiple myeloma, and GC019F, which targets adult B cell acute lymphoblastic leukemia. Both candidates are currently undergoing Phase I clinical trials. Additionally, the company is advancing GC007F for B cell non-Hodgkin's lymphoma and GC027 for adult T cell acute lymphoblastic leukemia, among others. Gracell also has a range of earlier-stage product candidates addressing various cancer types, such as ovarian cancer and breast cancer. Founded in 2017, the company aims to provide effective cellular therapeutics to improve outcomes for patients with hematological malignancies and solid tumors.

Passage Bio is a gene therapy company dedicated to developing transformative treatments for rare, monogenic central nervous system disorders. It has an integrated pipeline of five AAV-delivered therapeutics and collaborates with the University of Pennsylvania's Gene Therapy Program for preclinical work.

Burning Rock Biotech is a cancer diagnostics company that leverages next-generation sequencing and bioinformatics to guide personalized cancer treatment. It develops and provides a portfolio of tissue- and blood-based, NGS-driven therapy selection tests, including OncoScreen Plus, LungPlasma, ColonCore and HRDCore, and evaluates biomarkers such as microsatellite instability, tumor mutation burden, and NTRK fusions across multiple cancer types, including lung, gastrointestinal, prostate, breast, hematologic, thyroid, colorectal, ovarian, pancreatic, and bladder cancers. The company supports clinical and research use through one-stop services spanning genetic testing and companion diagnostic development, operating through central laboratory and in-hospital models. It collaborates with major pharmaceutical companies on clinical trials and research to advance targeted therapies and diagnostics. Founded in 2014, it is headquartered in Guangzhou, with a Guangzhou laboratory and an R&D center in Shanghai, reflecting its focus on expanding access to precision oncology in China.

EdiGene Inc. is a biotechnology company specializing in the development of genome editing technologies for therapeutic applications in cancer and various genetic diseases. Founded in 2015 and headquartered in Beijing, China, with additional offices in Guangzhou and Cambridge, Massachusetts, EdiGene focuses on both ex vivo and in vivo gene-editing therapies. The company has established proprietary platforms for editing hematopoietic stem cells and T cells, as well as an RNA base editing approach for in vivo applications. EdiGene's innovative methods enable targeted modifications of genetic information in human cells and facilitate high-throughput drug discovery through advanced screening techniques. By translating these technologies into novel therapeutics, EdiGene aims to improve the safety and efficacy of gene therapy, ultimately benefiting patients, the pharmaceutical industry, and the scientific research community.

Geneception is a company focused on the development of breakthrough in gene and cell therapies that are fully-integrated.

Dicerna Pharmaceuticals is a biopharmaceutical company that specializes in the discovery and development of RNA interference (RNAi)-based therapeutics. The company focuses on innovative treatments for rare inherited liver diseases, viral infections, chronic liver diseases, and cardiometabolic conditions. Utilizing its proprietary GalXC RNAi technology platform, Dicerna is advancing several key product candidates, including nedosiran for primary hyperoxaluria, RG6346 for chronic hepatitis B, and programs targeting genetic liver diseases and cardiovascular conditions. Founded in 2007 and headquartered in Lexington, Massachusetts, Dicerna has established strategic collaborations with major pharmaceutical firms to enhance its research and development efforts.

KSQ Therapeutics, Inc., founded in 2015 and based in Cambridge, Massachusetts, specializes in precision functional genomics to discover innovative drug therapies. The company employs a novel approach to research and development that emphasizes de-risking prior to the creation of therapeutic candidates. Utilizing its proprietary CRISPRomics™ drug discovery engine, KSQ Therapeutics has mapped the functions of human genes across various diseases, enhancing its understanding of disease biology. This comprehensive insight enables the identification and validation of high-confidence, patient-specific drug targets. As a result, the company is focused on developing medicines that have significant potential to improve patient outcomes and has established a pipeline of preclinical discovery programs.

EdiGene Inc. is a biotechnology company specializing in the development of genome editing technologies for therapeutic applications in cancer and various genetic diseases. Founded in 2015 and headquartered in Beijing, China, with additional offices in Guangzhou and Cambridge, Massachusetts, EdiGene focuses on both ex vivo and in vivo gene-editing therapies. The company has established proprietary platforms for editing hematopoietic stem cells and T cells, as well as an RNA base editing approach for in vivo applications. EdiGene's innovative methods enable targeted modifications of genetic information in human cells and facilitate high-throughput drug discovery through advanced screening techniques. By translating these technologies into novel therapeutics, EdiGene aims to improve the safety and efficacy of gene therapy, ultimately benefiting patients, the pharmaceutical industry, and the scientific research community.

IMPACT Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics for cancer and other life-threatening diseases. Founded in 2009 and based in Shanghai, China, the company focuses on creating targeted anti-cancer treatments utilizing synthetic lethality. IMPACT Therapeutics is actively engaged in several research and development projects, including a PARP inhibitor, a Wee1 inhibitor, and various novel agents targeting DNA damage response mechanisms and the Hedgehog signaling pathway. Through its commitment to advancing treatment options, IMPACT Therapeutics aims to provide patients with effective and targeted anti-cancer therapies.

Tmunity Therapeutics, Inc. is a biotherapeutics company based in Philadelphia, Pennsylvania, that specializes in the development of innovative immunotherapies for cancer, infectious diseases, and autoimmune disorders. Founded in 2015, Tmunity focuses on harnessing the potential of T cells through novel technologies, including T Cell Receptor (TCR) engineered T cells and regulatory T cells (Treg). The company has developed advanced platforms that allow for precise control over T cell activation and direction in vivo, as well as proprietary methods for activating, expanding, and genetically engineering T cells sourced from peripheral blood, cord blood, and tumors. Tmunity aims to advance personalized next-generation immunotherapies and has a team of experts with extensive experience in T-cell biology and clinical development, enhancing its capability to conduct safe and effective clinical trials. Its robust manufacturing platform supports multi-center clinical studies across various therapeutic areas, positioning Tmunity at the forefront of immunotherapy innovation.

Sigilon Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, dedicated to developing functional cures for patients with chronic diseases. Founded in 2015, the company aims to create immune-protected, engineered human cells that restore normal physiological functions without the risk of fibrosis or immune rejection. Its lead product candidate, SIG-001, is currently in Phase I/II clinical trials for preventing bleeding episodes in patients with moderate to severe Hemophilia A. Additionally, Sigilon is advancing other candidates, including SIG-005, which targets non-neurological manifestations of mucopolysaccharidosis type 1, and SIG-002, designed to replace islet cells for treating type 1 diabetes. The company's innovative Shielded Living Therapeutics platform leverages advances in cell engineering and proprietary biocompatible materials to offer stable and durable therapeutic solutions for a variety of chronic conditions.

Singlera Genomics Inc. is a biotechnology company that specializes in genetic testing and molecular diagnostic technologies. Founded in 2014 and headquartered in La Jolla, California, with additional operations in Shanghai, China, Singlera develops a methylation detection platform designed for early cancer diagnosis and prevention. The company offers a range of services, including tumor diagnosis, personalized treatment, non-invasive prenatal diagnosis, and pre-implantation genetic screening. Additionally, Singlera is focused on advancing precision medicine, aiming to provide patients with early, accurate, and informative diagnostic solutions to improve healthcare outcomes.

Tmunity Therapeutics, Inc. is a biotherapeutics company based in Philadelphia, Pennsylvania, that specializes in the development of innovative immunotherapies for cancer, infectious diseases, and autoimmune disorders. Founded in 2015, Tmunity focuses on harnessing the potential of T cells through novel technologies, including T Cell Receptor (TCR) engineered T cells and regulatory T cells (Treg). The company has developed advanced platforms that allow for precise control over T cell activation and direction in vivo, as well as proprietary methods for activating, expanding, and genetically engineering T cells sourced from peripheral blood, cord blood, and tumors. Tmunity aims to advance personalized next-generation immunotherapies and has a team of experts with extensive experience in T-cell biology and clinical development, enhancing its capability to conduct safe and effective clinical trials. Its robust manufacturing platform supports multi-center clinical studies across various therapeutic areas, positioning Tmunity at the forefront of immunotherapy innovation.

Sansure Biotech Inc. is a biotechnology company based in Changsha, Hunan Province, established in 2008. The company specializes in molecular diagnosis and genetic testing, developing and manufacturing a range of products that include nucleic acid extraction kits, real-time polymerase chain reaction (PCR) diagnostic kits, and fluorescence systems. Its product portfolio encompasses diagnostic kits for various health concerns, including novel coronavirus detection, viral hepatitis, AIDS, cancer biomarkers, and respiratory, digestive, and genetic diseases. Additionally, Sansure Biotech provides instruments and consumables to support its diagnostic offerings. The company primarily serves hospitals and laboratories both in China and internationally, focusing on enhancing diagnostic capabilities across multiple medical fields.

Geneception is a company focused on the development of breakthrough in gene and cell therapies that are fully-integrated.

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.

Cavion LLC is a clinical-stage pharmaceutical company focused on developing therapies that restore the brain’s natural rhythms by modulating T-type calcium channels (Cav3), which are implicated in various neurological and rare genetic diseases. The company, founded in 2003 and based in Charlottesville, Virginia, with an additional office in Cambridge, Massachusetts, has developed CX-8998, a drug that targets these calcium channels in the nervous system. Cavion's innovative approach also extends to cancer treatment, as the company was the first to create therapies that engage Cav3 for solid tumors, with preclinical successes leading to Phase 1 clinical trials in brain cancer. The company's neurology program aims to deliver a novel class of non-addictive, non-opioid T-type calcium channel antagonists for conditions like neuropathic pain, essential tremor, Parkinson’s disease, epilepsy, and Angelman syndrome. Cavion operates as a subsidiary of Jazz Pharmaceuticals plc, reflecting its commitment to advancing treatments that address critical medical needs in both oncology and neurology.

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.

Veritas Genetics International Ltd. is a genetic testing service provider based in Danvers, Massachusetts, with operations across North America, Asia, and Europe. The company collaborates with scientists, physicians, advisors, and patient advocates to develop and deliver innovative genetic testing solutions. Veritas Genetics utilizes advanced genetic technology that can identify a broader spectrum of gene variations than conventional next-generation sequencing methods. Among its offerings, the company provides a screening test for hereditary breast and ovarian cancer risk, facilitating informed decision-making for clinical researchers and healthcare providers. Through its services, Veritas Genetics aims to empower individuals to lead healthier and longer lives.

ReadCoor, Inc. is a technology company based in Cambridge, Massachusetts, specializing in the development and commercialization of a panomic spatial sequencing platform. The company offers the RC2 Platform, which utilizes proprietary Fluorescent in situ Sequencing (FISSEQ) technology to enable researchers, clinicians, and pharmaceutical and diagnostics companies to perform multi-omic analyses. This platform allows for the simultaneous detection and sequencing of numerous RNA, DNA, proteins, and therapeutics while visualizing sub-cellular nanoscale 3D resolution across tissue sections. By integrating high throughput sequencing with morphometric analysis and three-dimensional spatial imaging, ReadCoor provides valuable insights into human biology, facilitating the development of new therapeutics for various conditions, including cancer, infectious diseases, and neurological disorders. Founded in 2016, ReadCoor operates as a subsidiary of 10x Genomics, Inc. as of October 2020.

Singlera Genomics Inc. is a biotechnology company that specializes in genetic testing and molecular diagnostic technologies. Founded in 2014 and headquartered in La Jolla, California, with additional operations in Shanghai, China, Singlera develops a methylation detection platform designed for early cancer diagnosis and prevention. The company offers a range of services, including tumor diagnosis, personalized treatment, non-invasive prenatal diagnosis, and pre-implantation genetic screening. Additionally, Singlera is focused on advancing precision medicine, aiming to provide patients with early, accurate, and informative diagnostic solutions to improve healthcare outcomes.

IMPACT Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics for cancer and other life-threatening diseases. Founded in 2009 and based in Shanghai, China, the company focuses on creating targeted anti-cancer treatments utilizing synthetic lethality. IMPACT Therapeutics is actively engaged in several research and development projects, including a PARP inhibitor, a Wee1 inhibitor, and various novel agents targeting DNA damage response mechanisms and the Hedgehog signaling pathway. Through its commitment to advancing treatment options, IMPACT Therapeutics aims to provide patients with effective and targeted anti-cancer therapies.

Tmunity Therapeutics, Inc. is a biotherapeutics company based in Philadelphia, Pennsylvania, that specializes in the development of innovative immunotherapies for cancer, infectious diseases, and autoimmune disorders. Founded in 2015, Tmunity focuses on harnessing the potential of T cells through novel technologies, including T Cell Receptor (TCR) engineered T cells and regulatory T cells (Treg). The company has developed advanced platforms that allow for precise control over T cell activation and direction in vivo, as well as proprietary methods for activating, expanding, and genetically engineering T cells sourced from peripheral blood, cord blood, and tumors. Tmunity aims to advance personalized next-generation immunotherapies and has a team of experts with extensive experience in T-cell biology and clinical development, enhancing its capability to conduct safe and effective clinical trials. Its robust manufacturing platform supports multi-center clinical studies across various therapeutic areas, positioning Tmunity at the forefront of immunotherapy innovation.

Immunocore is a biotechnology company focused on developing novel T cell receptor-based therapies to treat diseases with high unmet needs, such as cancer and viral infections. Its proprietary platform enables the creation of bispecific immunotherapies, with IMCgp100 being its lead clinical candidate for metastatic melanoma.

Veritas Genetics International Ltd. is a genetic testing service provider based in Danvers, Massachusetts, with operations across North America, Asia, and Europe. The company collaborates with scientists, physicians, advisors, and patient advocates to develop and deliver innovative genetic testing solutions. Veritas Genetics utilizes advanced genetic technology that can identify a broader spectrum of gene variations than conventional next-generation sequencing methods. Among its offerings, the company provides a screening test for hereditary breast and ovarian cancer risk, facilitating informed decision-making for clinical researchers and healthcare providers. Through its services, Veritas Genetics aims to empower individuals to lead healthier and longer lives.

IMPACT Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapeutics for cancer and other life-threatening diseases. Founded in 2009 and based in Shanghai, China, the company focuses on creating targeted anti-cancer treatments utilizing synthetic lethality. IMPACT Therapeutics is actively engaged in several research and development projects, including a PARP inhibitor, a Wee1 inhibitor, and various novel agents targeting DNA damage response mechanisms and the Hedgehog signaling pathway. Through its commitment to advancing treatment options, IMPACT Therapeutics aims to provide patients with effective and targeted anti-cancer therapies.

Crown Bioscience International is a drug discovery and preclinical research company that provides translational platforms and services to biotech and pharma firms. Its offerings span oncology, cardiovascular, and metabolic disease, including in vivo and in vitro antibodies, bioactive recombinant proteins, recombinant cell lines, tumor cells, immune checkpoint antibodies, and tumor tissue microarrays. The company provides integrated preclinical oncology services and protein science capabilities such as cloning, expression, purification, protein assays, and structural biology to accelerate drug development. It supports evaluation of efficacy, pharmacology, and patient response profiles to improve clinical decision-making and patient stratification. Crown Bioscience collaborates with external centers to license and commercialize genetically engineered models and to deliver organoid-based oncology preclinical development. Founded in 2006 and based in Taipei, Taiwan, Crown Bioscience operates globally through multiple facilities and partnerships to serve biotech and pharmaceutical customers.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

Forma Therapeutics, headquartered in Watertown, Massachusetts, is a clinical-stage biopharmaceutical company focused on developing and commercializing innovative therapeutics for rare hematologic diseases and various cancers. The company is advancing several key product candidates, including FT-4202, currently in Phase 1 trials for sickle cell disease and other hemoglobinopathies, and FT-7051, targeting metastatic castration-resistant prostate cancer. Additionally, Forma is developing FT-2102, a selective small molecule designed to inhibit mutated IDH1 enzymes, which is being evaluated in Phase 2 trials for relapsed/refractory acute myeloid leukemia and in exploratory trials for glioma. Other candidates include FT-4101 and FT-8225, selective inhibitors of fatty acid synthase. The firm integrates advanced drug discovery technologies with deep oncology expertise to address challenging targets, contributing to a robust pipeline aimed at transforming patient outcomes in oncology.

Intradigm is a private biotechnology company specializing in the development of systematic RNA interference (RNAi) therapeutics aimed at treating serious diseases, particularly in the field of oncology. The company leverages its expertise in drug development and delivery to create an innovative RNAi platform. Central to its approach is the proprietary RNAi Nanoplex delivery technology, which allows for the targeted systemic delivery of RNAi therapeutics to specific tissues. Intradigm's portfolio includes a range of siRNA sequences designed to address various oncology and other disease targets, complemented by biodegradable polycationic polymers that enhance the delivery of these therapeutics.

ImClone Systems is a prominent biotechnology company focused on the development and manufacture of therapeutic products aimed at improving oncology care. Established in 1984, the company specializes in creating targeted biologic treatments for various cancers, leveraging advancements in molecular biology, genomics, and antibody engineering. Its flagship product, ERBITUX, is complemented by a diverse pipeline of investigational monoclonal antibodies currently in mid- to late-stage clinical development, targeting major solid tumor types. Following its acquisition by Eli Lilly and Company in 2008, ImClone has enhanced its pipeline development through Lilly's global resources. The company operates state-of-the-art FDA-approved manufacturing facilities in Branchburg, New Jersey, which are among the largest for biologic manufacturing worldwide. Additionally, ImClone has research headquarters in New York City and maintains international operations in Europe, positioning it well to meet the evolving medical needs of cancer patients.

As of August 20, 2008, SGX Pharmaceuticals, Inc. was acquired by Eli Lilly & Co. SGX Pharmaceuticals, Inc., a biotechnology company, focuses on the discovery, development, and commercialization of therapeutics for addressing unmet medical needs in oncology. The company's product pipeline includes drug candidates from its FAST drug discovery platform, which uses X-ray crystallography and complementary biophysical and biochemical methods, combined with medicinal and computational chemistry. Its drug development programs target the c-MET receptor tyrosine kinase (MET), an enzyme implicated in an array of cancers; and the BCR-ABL tyrosine kinase enzyme for the treatment of chronic myelogenous leukemia (CML), a bone marrow cancer. Under the MET development program, the company develops SGX523, which is in Phase I clinical development studies for solid tumor indications; and SGX126, a preclinical development product for solid tumors indications. Under the BCR-ABL Development Program, SGX Pharmaceuticals develops SGX393, an oral therapy for the second-line treatment of CML, which is in preclinical development studies. Its drug discovery technologies are also applied to a portfolio of oncology targets, including JAK2, a non-receptor tyrosine kinase involved in cytokine-induced signaling and growth regulation, survival, and differentiation of cells; RAS, a protein that regulates cell growth; and three other tyrosine kinases. The company has a license and collaboration agreement with Novartis Institutes for Biomedical Research, Inc., Cystic Fibrosis Foundation Therapeutics, Inc., National Institutes of Health, and Eli Lilly & Company. SGX Pharmaceuticals, formerly known as Structural GenomiX, Inc., was founded in 1998 and is headquartered in San Diego, California.

Standard BioTools develops, manufactures, and sells technologies that accelerate biomedical research and medicines development. The company provides standardized, next-generation tools built around proprietary mass cytometry and microfluidics platforms to generate reliable insights into health and disease. Its proteomics and genomics capabilities support a range of applications in translational and clinical research, including oncology, immunology, and immunotherapy. The products are used by academic, government, pharmaceutical, biotechnology, plant and animal research, and clinical laboratories worldwide. By delivering repeatable measurement platforms, Standard BioTools helps researchers translate discoveries into improved patient outcomes.

CGI Pharmaceuticals, founded in 2000 from Yale University, specializes in the discovery and development of small molecule therapeutics targeting oncology and allergy/autoimmune/inflammatory diseases. The company employs a distinctive chemical-genetics approach known as ASKA (Analog Sensitive Kinase Alleles) to investigate kinase function, collaborating with various biotech and pharmaceutical partners. CGI has developed a proprietary library of over 50,000 small molecule kinase inhibitors, all designed and synthesized in-house. Its comprehensive drug discovery infrastructure includes expertise in kinase biology, chemistry, lead generation, and drug metabolism/pharmacokinetics (DMPK). CGI has been granted eight U.S. patents for its innovative kinase inhibitors, and all of its drug discovery and development programs are derived from candidates identified within its proprietary library. Notable programs focus on Btk, a B-cell kinase for multiple indications, and Syk, which regulates the functions of mast cells, monocytes, macrophages, and B-cells.

Perlegen Sciences engages in the discovery and commercialization of genetic variations that provide physicians with information to improve the clinical decision-making process and patient treatment outcomes. Its products include MammaPLUS, a breast cancer risk stratifier and a panel of common genetic variants that stratifies individual genetic and background/clinical risk for those at moderately elevated risk for non-familial breast cancer, as well as impacts the risk for developing invasive breast cancer; and Genotype Browser Website that provides a way to access and view the results of a study of genetic variation in various diverse human populations. Perlegen Sciences, Inc. was founded in 2000 and is based in Mountain View, California.