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Atsena Therapeutics is a clinical-stage company focused on developing gene therapies for inherited retinal diseases. Utilizing an adeno-associated virus (AAV) platform, including a novel spreading capsid, Atsena's clinical programs aim to prevent blindness caused by genetic mutations.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

Kate Therapeutics is a biotechnology company specializing in developing adeno-associated virus (AAV)-based gene therapies for genetically defined muscle and heart diseases. It employs innovative technology platforms to enhance tissue-specific delivery and gene regulation, addressing key limitations of existing gene therapies.

Chroma Medicine is a genomic medicine company founded in 2021 and based in Cambridge, Massachusetts, specializing in epigenetic editing. The company is pioneering a new class of genomic therapies aimed at revolutionizing the treatment of genetically driven diseases. By utilizing epigenetics, which is the natural mechanism for gene regulation, Chroma Medicine develops programmable epigenetic editors that target specific genes and modulate chromatin conformation. This innovative approach enables healthcare providers to achieve precise control of gene expression, offering a promising therapeutic option for conditions linked to genetic anomalies.

Delfi Diagnostics, Inc. is a biotechnology company based in Baltimore, Maryland, focused on the early detection of cancer through innovative blood tests. Founded in 2018, the company employs artificial intelligence and genome sequencing to identify unique patterns of DNA fragmentation in the blood, enabling the detection of previously unrecognized cancer-associated cell-free DNA fragments. By utilizing machine learning, Delfi Diagnostics aims to develop high-precision, non-invasive blood tests that allow healthcare professionals to detect cancer at its most curable stage, thereby facilitating timely and effective treatment options for patients.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

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.

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.

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

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Delfi Diagnostics, Inc. is a biotechnology company based in Baltimore, Maryland, focused on the early detection of cancer through innovative blood tests. Founded in 2018, the company employs artificial intelligence and genome sequencing to identify unique patterns of DNA fragmentation in the blood, enabling the detection of previously unrecognized cancer-associated cell-free DNA fragments. By utilizing machine learning, Delfi Diagnostics aims to develop high-precision, non-invasive blood tests that allow healthcare professionals to detect cancer at its most curable stage, thereby facilitating timely and effective treatment options for patients.

Atsena Therapeutics is a clinical-stage company focused on developing gene therapies for inherited retinal diseases. Utilizing an adeno-associated virus (AAV) platform, including a novel spreading capsid, Atsena's clinical programs aim to prevent blindness caused by genetic mutations.

Palladio Biosciences, Inc. is a biotechnology company focused on developing innovative medicines for orphan kidney diseases. Established in 2015 and headquartered in Horsham, Pennsylvania, the company is primarily engaged in creating therapeutics for conditions such as polycystic kidney disease (PKD), a serious genetic disorder characterized by the formation of fluid-filled cysts in the kidneys. One of its key products, Lixivaptan, is a selective vasopressin V2 receptor antagonist aimed at treating PKD, helping to prevent disease progression and improve patient outcomes. Palladio Biosciences is dedicated to addressing unmet medical needs in the field of renal diseases.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

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.

Atsena Therapeutics is a clinical-stage company focused on developing gene therapies for inherited retinal diseases. Utilizing an adeno-associated virus (AAV) platform, including a novel spreading capsid, Atsena's clinical programs aim to prevent blindness caused by genetic mutations.

ReCode Therapeutics, Inc. is a biopharmaceutical company focused on developing precision medicines for pulmonary diseases, particularly targeting genetic conditions such as primary ciliary dyskinesia and cystic fibrosis. Founded in 2015 and based in Menlo Park, California, the company utilizes a groundbreaking selective organ targeting (SORT) lipid nanoparticle platform. This innovative technology facilitates the precise delivery of genetic therapies, including mRNA and gene correction therapeutics, to specific organs and cells beyond the liver. By overcoming limitations of traditional gene therapy approaches, ReCode Therapeutics aims to address the needs of patients with rare and life-limiting respiratory diseases, thus providing new treatment options for those affected by genetic disorders.

ArsenalBio is a programmable cell therapy company established in 2019, focused on developing and commercializing innovative immune cell therapies, particularly for cancer treatment. The company utilizes advanced technologies, including CRISPR-based genome engineering, synthetic biology, and machine learning, to create multifunctional T-cell therapies. ArsenalBio's approach involves the precise insertion of large synthetic DNA sequences, enabling the generation of next-generation autologous T-cell therapies. By integrating these cutting-edge methods, ArsenalBio aims to enhance the effectiveness and safety of immune cell therapies, reduce costs for healthcare providers, and improve market access, ultimately broadening patient outcomes and making these therapies more widely available.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Twentyeight-Seven Therapeutics is a developer of microRNAs designed to target regulatory proteins.The company's modulating RNA which are short non-coding RNAs that inhibit target gene expression by suppressing mRNA translation and promoting mRNA decay and develop small molecules that can modulate levels of miRNAs by targeting proteins that interact with these miRNAs, enabling users to target miRNAs are directly involved in cancer initiation, progression, and metastasis.

LunaDNA is a community-owned genomic and medical research platform founded by the creators of a leading DNA sequencing company. It facilitates the sharing of health and DNA data, allowing individuals to contribute their personal information for medical research while maintaining ownership of their data. By incentivizing participation, LunaDNA addresses the challenge of data silos that have emerged as personal DNA sequencing has gained popularity. Participants are rewarded with shares in the database, granting them a stake in the profits generated from medical research. These profits are returned to the community as dividends when researchers pay for access to the data. LunaDNA empowers individuals to support health research while ensuring that their contributions benefit the broader community and drive medical discoveries.

LunaPBC, Inc. is a Public Benefit Corporation based in San Diego, California, focused on developing a community-owned platform for health and DNA research. The company's primary offering is LunaDNA, a genomic and medical research knowledge database that enables individuals to share their health and DNA data for research purposes. This platform facilitates collaborations with pharmaceutical companies, insurance providers, and healthcare IT organizations to drive advancements in medical discoveries. By allowing members to contribute their health data, LunaPBC not only supports vital health research projects but also ensures that participants can share in the financial benefits arising from medical breakthroughs, fostering a sense of community ownership and engagement in the research process.

Arrakis Therapeutics, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, that specializes in drug discovery focused on ribonucleic acid (RNA) targets. Established in 2015, the company utilizes a proprietary platform that combines advanced RNA bioinformatics, structural tools, and chemical libraries to identify new RNA targets and develop RNA-targeted small molecules (rSMs). This innovative approach aims to address various diseases, including cancer, neurological disorders, and rare genetic conditions. Arrakis also offers SHAPEware, a software tool designed to analyze and predict RNA secondary structures and potential ligand-binding sites, further enhancing its drug development capabilities. Through its comprehensive pipeline, Arrakis Therapeutics seeks to improve treatment options and outcomes for patients across multiple therapeutic areas.

Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases characterized by high unmet medical needs. Incorporated in 2016 and based in Oakland, California, the company is known for its lead therapy, MT1621, which aims to restore mitochondrial DNA replication fidelity. This therapy specifically targets thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disorder. By improving nucleotide balance and increasing mitochondrial DNA copy number, Modis Therapeutics' approach seeks to enhance cellular function and prolong the lives of affected patients. As of September 2019, Modis Therapeutics operates as a subsidiary of Zogenix, Inc.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Medisix Therapeutics, established in 2016 and headquartered in Singapore, is an early-stage biotechnology company specializing in cell therapy. It focuses on developing chimeric antigen receptor (CAR) T-cell therapies to target and treat T-cell lymphomas and leukemias. The company's technology platform is built upon the pioneering work of Professor Dario Campana, MD, PhD, a renowned expert in translational immunology and the founder of CAR-T cell biology. Medisix's proprietary immune engineering approaches enable the targeting of T cell leukemias and lymphomas using cell therapy.

Beam Therapeutics is a biotechnology company developing precision genetic medicines using its proprietary base editing technology. Its focus includes therapies for hematological and genetic diseases, with ongoing programs targeting sickle cell disease, alpha-1 antitrypsin deficiency, and other serious conditions.

Synthorx, Inc. is a biopharmaceutical company specializing in the development of innovative protein therapeutics using advanced synthetic biology techniques. Founded in 2014 and headquartered in La Jolla, California, Synthorx focuses on its proprietary Synthorin programs aimed at treating cancer and autoimmune disorders. Its lead product candidate, THOR-707, is a modified form of recombinant human IL-2, currently being evaluated for various solid tumors both as a standalone treatment and in combination with immune checkpoint inhibitors. The company also develops Synthorins targeting IL-2 for autoimmune conditions, IL-10 for immuno-oncology applications, and IL-15 for regulating immune responses. Synthorx leverages a unique platform that enables the incorporation of synthetic DNA base pairs to enhance the specificity and efficacy of its protein therapeutics, thus offering improved treatment options for patients. Previously known as Alinos, Inc., the company acquired exclusive rights to its synthetic biology technology based on groundbreaking research from The Scripps Research Institute. As of January 2020, Synthorx operates as a subsidiary of Sanofi.

Cyteir Therapeutics, Inc. is a clinical-stage biotechnology company dedicated to developing innovative therapies based on DNA repair biology for cancer treatment and synthetic lethality in autoimmune diseases. Founded in 2012 and headquartered in Lexington, Massachusetts, the company focuses on precision targeting of critical DNA repair factors to induce self-destruction in diseased cells through an overload of DNA damage. Cyteir employs an integrated drug development platform that addresses the imbalance between DNA damage and repair, resulting in a pipeline of small molecule therapeutics aimed at various disease states, including hematological malignancies, solid tumors, and chronic autoimmune disorders. Its lead compound, CYT-0851, is an oral investigational drug designed to inhibit RAD51-mediated homologous recombination, targeting specific vulnerabilities in cancer cells.

Homology Medicines is a biotechnology company focused on developing genetic therapies for rare diseases. Its proprietary platform uses human hematopoietic stem cell-derived adeno-associated virus vectors to deliver treatments in vivo, targeting various tissues including the liver and central nervous system. The company's lead product candidate, HMI-102, is in clinical trials for treating phenylketonuria.

ESCAPE Bio is a clinical-stage biopharmaceutical company focused on discovering and developing small molecule therapies for genetically defined subpopulations affected by neurodegenerative diseases. Founded in 2015 and based in South San Francisco, California, the company aims to improve the lives of patients suffering from conditions such as Alzheimer's disease, Parkinson's disease, and sphingolipid storage disorders. ESCAPE Bio's pipeline includes innovative candidates like ESB1609, a small molecule S1P5 receptor agonist targeting CNS lysosomal storage disorders, and ESB5070, a kinase inhibitor specifically designed for Parkinson's patients with the LRRK2 G2019S variant. Additionally, the company is developing a program aimed at addressing the needs of Alzheimer's patients who carry the ApoE4 risk allele. Through its precision-targeted approach, ESCAPE Bio seeks to provide effective treatment options for individuals with these complex genetic profiles.

Arrakis Therapeutics, Inc. is a biopharmaceutical company based in Waltham, Massachusetts, that specializes in drug discovery focused on ribonucleic acid (RNA) targets. Established in 2015, the company utilizes a proprietary platform that combines advanced RNA bioinformatics, structural tools, and chemical libraries to identify new RNA targets and develop RNA-targeted small molecules (rSMs). This innovative approach aims to address various diseases, including cancer, neurological disorders, and rare genetic conditions. Arrakis also offers SHAPEware, a software tool designed to analyze and predict RNA secondary structures and potential ligand-binding sites, further enhancing its drug development capabilities. Through its comprehensive pipeline, Arrakis Therapeutics seeks to improve treatment options and outcomes for patients across multiple therapeutic areas.

Precision BioSciences, founded in 2006, is a biotechnology company headquartered in Durham, North Carolina. It specializes in genome editing, developing therapeutic products using its proprietary ARCUS technology. The company focuses on creating allogeneic CAR T immunotherapies to treat cancers like acute lymphoblastic leukemia and non-hodgkin lymphoma, as well as genetic disorders.

Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.

Guardant Health is a precision oncology company specializing in blood tests and analytics. It provides liquid biopsy tests like Guardant360 for advanced cancer detection, GuardantOMNI for broader gene panels, LUNAR-1 for minimal residual disease detection, and LUNAR-2 for early cancer detection. The company also offers development services to biopharmaceutical companies.

Applied Genetic Technologies Corporation is a clinical-stage biotechnology company focused on developing innovative genetic therapies for patients with rare and debilitating diseases. The company has advanced product candidates in ophthalmology, including treatments for X-linked retinitis pigmentosa and achromatopsia, with the former having completed Phase I/II trials and the latter currently in the same stage. Additionally, AGTC is working on an optogenetic therapy for patients with advanced retinal diseases. Beyond ophthalmology, the company has initiated preclinical programs targeting otology and central nervous system disorders, including frontotemporal dementia, amyotrophic lateral sclerosis, and adrenoleukodystrophy. AGTC employs gene therapy techniques to replace defective genes with functional ones, aiming to provide long-lasting treatment solutions through single injections. Established in 1999 and headquartered in Alachua, Florida, the company collaborates with various academic and industry partners to enhance its research and development efforts.

Mirna Therapeutics, Inc. is a discovery-stage biopharmaceutical company that specializes in developing miRNA-directed therapies for human oncology. With a strong focus on research and a deep understanding of microRNA and cancer biology, Mirna is positioned to advance the emerging field of miRNA-based therapeutics. The company boasts a robust intellectual property portfolio that supports its innovative approach to cancer treatment.