Eli Lilly and Company

ProQR Therapeutics N.V. is a biopharmaceutical company based in Leiden, the Netherlands, focused on the discovery and development of RNA-based therapeutics for genetic disorders. Founded in 2012, the company targets severe rare diseases, including Leber's congenital amaurosis type 10 and Usher syndrome type 2. ProQR's leading product candidates include sepofarsen, currently in a phase II/III clinical trial for Leber's congenital amaurosis 10, QR-421a in a phase 1/2 trial for Usher syndrome type 2 and non-syndromic retinitis pigmentosa, QR-1123 in a phase 1/2 trial for autosomal dominant retinitis pigmentosa, and QR-504a, which is undergoing its first clinical trial for Fuchs endothelial corneal dystrophy. The company has established license agreements with several prominent medical institutions, including General Hospital Corporation and Radboud University Medical Center.

OrsoBio is a clinical-stage biopharmaceutical company focused on developing innovative therapies to treat severe metabolic disorders such as obesity, type 2 diabetes, lipodystrophies, and severe dyslipidemia. The company aims to restore energy homeostasis in patients by targeting pathways relevant to maintaining energy balance, thereby addressing the root causes of these conditions. OrsoBio's pipeline includes therapies intended to revolutionize the treatment of these disorders.

HAYA Therapeutics is a company focused on developing RNA-based therapeutics aimed at treating heart failure and other fibrotic diseases. The company specializes in biopharmaceutical therapies that target specific regulators of fibrosis, particularly long noncoding RNA, which plays a critical role in myocardial fibrosis. By honing in on heart-specific mechanisms, HAYA Therapeutics seeks to enhance the efficacy and safety of treatments for heart-related illnesses and other serious conditions associated with aging, including cancer. Through its innovative approach, the company aims to improve patient outcomes in the management of heart failure and related health challenges.

Ajax Therapeutics is a biotechnology company based in New York City that focuses on developing innovative small molecule therapies for hematologic malignancies. The company utilizes computational chemistry and structure-based technologies to design drugs that target critical cytokine signaling pathways involved in these cancers. By integrating insights from disease biology, genetics, and structural biology with an advanced computational drug discovery platform, Ajax Therapeutics aims to create precisely designed therapeutics that address significant unmet medical needs for patients suffering from hematologic malignancies.

OrsoBio is a clinical-stage biopharmaceutical company focused on developing innovative therapies to treat severe metabolic disorders such as obesity, type 2 diabetes, lipodystrophies, and severe dyslipidemia. The company aims to restore energy homeostasis in patients by targeting pathways relevant to maintaining energy balance, thereby addressing the root causes of these conditions. OrsoBio's pipeline includes therapies intended to revolutionize the treatment of these disorders.

Laverock Therapeutics is focused on advancing a gene silencing platform that facilitates the development of programmable, allogeneic cell therapies. This innovative platform allows for the engineering of next-generation cell therapies that are both adaptive and capable of responding to various intra- and extra-cellular environmental cues. By enhancing the functionality of these therapies, Laverock Therapeutics aims to improve the efficacy, safety, and accessibility of iPSC-derived products, addressing significant limitations associated with current therapeutic approaches. The company's work is positioned to benefit medical and biotechnology institutions seeking to leverage advanced cell therapy solutions.

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

Verve Therapeutics is a genetic medicines company based in Cambridge, Massachusetts, focused on innovative therapies for cardiovascular disease. Founded in 2018, the company aims to transform the management of cardiovascular conditions from chronic treatments to single-course gene editing medicines. Verve Therapeutics employs advanced gene-editing technology and human genetic analysis to develop targeted therapies that reduce the risk of coronary artery diseases. Its initial programs focus on the PCSK9 and ANGPTL3 genes, which are crucial for lowering harmful blood lipids like low-density lipoprotein cholesterol. The company collaborates with strategic partners, including Beam Therapeutics for delivery technologies and Verily for gene editing delivery vehicles, enhancing its capabilities in the biotechnology and healthcare sectors.

ProQR Therapeutics N.V. is a biopharmaceutical company based in Leiden, the Netherlands, focused on the discovery and development of RNA-based therapeutics for genetic disorders. Founded in 2012, the company targets severe rare diseases, including Leber's congenital amaurosis type 10 and Usher syndrome type 2. ProQR's leading product candidates include sepofarsen, currently in a phase II/III clinical trial for Leber's congenital amaurosis 10, QR-421a in a phase 1/2 trial for Usher syndrome type 2 and non-syndromic retinitis pigmentosa, QR-1123 in a phase 1/2 trial for autosomal dominant retinitis pigmentosa, and QR-504a, which is undergoing its first clinical trial for Fuchs endothelial corneal dystrophy. The company has established license agreements with several prominent medical institutions, including General Hospital Corporation and Radboud University Medical Center.

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.

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, Inc. is a biotechnology company dedicated to developing precision genetic medicine aimed at restoring, improving, and preserving hearing for individuals with various forms of hearing loss. Utilizing a proprietary adeno-associated viral vector library and an innovative delivery approach, Akouos focuses on gene therapies tailored to genetically-defined patient populations, including those affected by specific genetic mutations, ototoxic drug exposure, and age-related hearing loss. The company's lead product candidate, AK-OTOF, targets hearing loss associated with mutations in the OTOF gene. Founded in 2016 and based in Boston, Massachusetts, Akouos collaborates with strategic partners, including Massachusetts Eye and Ear and Lonza, Inc., to advance its mission of providing effective treatments for sensorineural hearing loss.

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.

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.

Jaguar Gene Therapy is a clinical-stage biotechnology company focused on accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases. The company specializes in developing treatments that target nonworking or missing genes by introducing healthy copies back into the body to restore function. This approach aims to address significant unmet needs in healthcare by expediting the development of gene therapies from research to clinical application as safely and quickly as possible.

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.

Nido Biosciences, Inc., incorporated in 2018 and based in Boston, Massachusetts, is a clinical-stage company focused on developing precision medicines for severe neurological diseases. The company employs a functional genomics discovery platform utilizing human cell lines to identify novel therapeutic targets for various neuromuscular and neurodegenerative conditions. By leveraging advances in neuroscience and human genetics, Nido Biosciences aims to create precise medications that address the underlying biology of these diseases, ultimately restoring healthy cell function and improving patient outcomes.

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.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

Avidity Biosciences, Inc. is a biopharmaceutical company based in La Jolla, California, specializing in the development of oligonucleotide-based therapies, particularly through its innovative Antibody Oligonucleotide Conjugates (AOC) platform. This technology aims to address the limitations of traditional oligonucleotide therapies by combining the tissue selectivity of monoclonal antibodies with the precision of oligonucleotide treatments. Avidity's lead product candidate, AOC 1001, targets myotonic dystrophy type 1, a rare genetic muscle disease. In addition to this, the company is advancing multiple programs focused on muscle-related conditions, including muscle atrophy, Duchenne muscular dystrophy, facioscapulohumeral muscular dystrophy, and Pompe disease. Furthermore, Avidity is expanding its research to include immune and other cell types, positioning itself at the forefront of biopharmaceutical innovation for serious diseases.

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.

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.

MacroGenics, Inc. is a biopharmaceutical company based in Rockville, Maryland, specializing in the discovery and development of antibody-based therapeutics for cancer treatment. Founded in 2000, the company focuses on immuno-oncology and has a diverse pipeline of product candidates. Key developments include Margetuximab, a monoclonal antibody in Phase III clinical trials targeting HER2-expressing tumors, and Flotetuzumab, a DART molecule aimed at treating acute myeloid leukemia. Other investigational products include MGA012, MGD013, MGD019, and Enoblituzumab, which target various immune checkpoints and tumor-associated antigens. MacroGenics is also advancing combination therapies and antibody-drug conjugates, such as MGC018, which targets solid tumors expressing B7-H3. The company has formed strategic collaborations with notable partners, enhancing its research and development efforts in the biopharmaceutical landscape.

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.

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.

HAYA Therapeutics is a company focused on developing RNA-based therapeutics aimed at treating heart failure and other fibrotic diseases. The company specializes in biopharmaceutical therapies that target specific regulators of fibrosis, particularly long noncoding RNA, which plays a critical role in myocardial fibrosis. By honing in on heart-specific mechanisms, HAYA Therapeutics seeks to enhance the efficacy and safety of treatments for heart-related illnesses and other serious conditions associated with aging, including cancer. Through its innovative approach, the company aims to improve patient outcomes in the management of heart failure and related health challenges.