Blackstone Life Sciences

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Uniquity Bio is a clinical-stage drug development company dedicated to advancing innovative therapies, particularly in the fields of immunology and inflammation. The company is focused on identifying and developing novel therapeutics that aim to deliver transformative benefits to patients suffering from conditions with limited or no treatment options. Leveraging the expertise of seasoned pharmaceutical executives, Uniquity Bio effectively drives its research and development initiatives. With strong backing from notable investors, the company is well-positioned to fulfill its mission of delivering groundbreaking medicines to the healthcare market.

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

Amicus Therapeutics, Inc. is a biotechnology company dedicated to the discovery, development, and commercialization of therapies for rare and orphan diseases. Founded in 2002 and headquartered in Cranbury, New Jersey, the company focuses on developing innovative treatments, including Galafold, an orally administered pharmacological chaperone for Fabry disease. Amicus is also advancing its pipeline with a Phase 3 clinical study of AT-GAA for Pompe disease. The company's research includes a range of first-in-class small molecules designed to treat lysosomal storage diseases by stabilizing misfolded enzymes in patients. This therapeutic approach potentially enhances the effectiveness of enzyme replacement therapies. Amicus Therapeutics has established collaborations with institutions such as Nationwide Children's Hospital and the University of Pennsylvania to further its research and development efforts.

Sutro Biopharma, Inc. is a clinical-stage drug discovery, development, and manufacturing company based in South San Francisco, California. The company specializes in creating next-generation protein therapeutics for cancer and autoimmune disorders using its proprietary integrated cell-free protein synthesis platform known as XpressCF. Sutro Biopharma’s product candidates include STRO-001, an antibody-drug conjugate targeting CD74 for patients with multiple myeloma and non-Hodgkin lymphoma, and STRO-002, an antibody-drug conjugate directed against folate receptor-alpha for treating ovarian and endometrial cancers. The company has also established collaboration and licensing agreements to advance bispecific antibodies and antibody-drug conjugates primarily in the field of immuno-oncology. Founded in 2003, Sutro Biopharma is dedicated to developing innovative biopharmaceutical solutions.

PTC Therapeutics, Inc. is a biopharmaceutical company dedicated to the discovery, development, and commercialization of orally administered small-molecule therapeutics for the treatment of rare disorders and cancer. The company offers Translarna, used for nonsense mutation Duchenne muscular dystrophy in ambulatory patients, and Emflaza, a corticosteroid for the same condition. PTC Therapeutics is advancing several candidates in clinical development, including Translarna for other rare genetic conditions, RG7916 and RO7034067 for spinal muscular atrophy, and PTC596 and PTC299, which target cancer through dihydrooratate dehydrogenase inhibition. Additionally, the company is developing a gene therapy candidate, PTC-AADC, for Aromatic L-amino acid decarboxylase deficiency. PTC Therapeutics collaborates with various organizations, including F. Hoffman-La Roche Ltd and Massachusetts General Hospital, to further its research and commercialization efforts. Founded in 1998, the company is headquartered in South Plainfield, New Jersey.

Sanofi Oncology, a division of global healthcare company Sanofi, focuses on developing and delivering innovative treatments for various types of cancer. With a rich heritage in oncology, Sanofi Oncology leverages its extensive knowledge base to research and treat cancer from multiple angles, addressing diverse patient needs and disease stages. The company's portfolio includes therapies for rare diseases, immunology, and cardiovascular conditions, among others. Sanofi's commitment to patients drives its strategy to create long-term value through innovative treatments, from research and development to manufacturing and marketing.

Harmony Biosciences, established in 2017 and based in Plymouth Meeting, Pennsylvania, is a biopharmaceutical company focused on developing and commercializing therapies for patients with rare neurological disorders. Its primary product, WAKIX (pitolisant), is a first-in-class medication designed to increase histamine signaling in the brain, approved for treating cataplexy in adult narcolepsy patients. Harmony Biosciences operates as a subsidiary of Harmony Biosciences Holdings, Inc., which is dedicated to addressing unmet medical needs in this therapeutic area.

Talaris Therapeutics, Inc. is a biotechnology company focused on developing innovative cell therapies aimed at eliminating chronic immunosuppression for organ transplant recipients. The company's leading product, FCR001, is designed to enable living donor kidney transplant recipients to discontinue all chronic immunosuppression therapy within twelve months post-transplant. In addition to its applications in organ transplantation, Talaris’s technology facilitates the safe use of hematopoietic stem cells from unmatched and unrelated donors for bone marrow transplants, reducing the associated toxicity and risks of traditional methods. Founded in 1988 and originally named Regenerex, Inc., Talaris Therapeutics rebranded in March 2019. The company is headquartered in Louisville, Kentucky, with an additional office in Wellesley, Massachusetts.

Annexon Biosciences is a clinical-stage biopharmaceutical company focused on developing novel therapies for patients with classical complement-mediated disorders affecting the body, brain, and eye. The company's pipeline is built on a platform technology that targets well-researched autoimmune and neurodegenerative disease processes triggered by the aberrant activation of C1q, the initiating molecule of the classical complement pathway. Annexon's lead product candidate, ANX005, is a full-length monoclonal antibody designed for intravenous administration to treat autoimmune and neurodegenerative disorders. Another key candidate, ANX007, is a monoclonal antibody Fab formulated for intravitreal administration to address neurodegenerative ophthalmic disorders. The company employs a biomarker-driven development strategy to ensure that its product candidates effectively engage the target at therapeutic doses within the intended patient tissue, while also exploring additional orphan and large market indications.

Medtronic is a global leader in healthcare technology, specializing in innovative solutions for complex medical conditions. The company offers a wide range of treatments and therapies, including patient monitoring systems, robotic surgery, insulin pumps, and cardiac devices, catering to various health areas such as lungs, brain, diabetes, heart, and orthopedics. Medtronic's extensive portfolio addresses 70 different medical conditions, aiming to improve and extend the lives of patients worldwide.

Reata Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company based in Plano, Texas, that specializes in developing novel therapeutics for serious and life-threatening diseases. The company targets molecular pathways involved in cellular metabolism and inflammation, with a focus on conditions such as chronic kidney disease (CKD), pulmonary arterial hypertension, and neurodegenerative diseases. Reata's pipeline includes bardoxolone methyl, which is undergoing Phase III trials for CKD caused by Alport syndrome and for pulmonary arterial hypertension associated with connective tissue disease. Additionally, omaveloxolone is in Phase II trials for Friedreich’s ataxia. The company is also exploring RTA 901 for neurological conditions and has RORgT inhibitors in preclinical development for autoimmune and inflammatory diseases. Reata has established strategic collaborations for the development and commercialization of its therapies in various Asian markets and with AbbVie for certain Nrf2 activators. Founded in 2002 and rebranded in 2005, Reata Pharmaceuticals is committed to advancing innovative treatments for complex medical challenges.

Alnylam Pharmaceuticals is a biopharmaceutical company specializing in the discovery, development, and commercialization of RNA interference (RNAi) therapeutics. The company focuses on addressing rare genetic diseases, cardio-metabolic conditions, hepatic infectious diseases, and central nervous system/ocular disorders. Its marketed products include ONPATTRO, a treatment for hereditary transthyretin-mediated amyloidosis, and GIVLAARI, which targets acute hepatic porphyria. Alnylam’s pipeline includes investigational therapies such as givosiran for acute hepatic porphyria, lumasiran for primary hyperoxaluria type 1, and vutrisiran for transthyretin amyloidosis. The company has established strategic alliances with notable partners like Sanofi Genzyme and Regeneron Pharmaceuticals, and it collaborates with Vir Biotechnology on RNAi therapeutics for coronavirus infections. Founded in 2002 and headquartered in Cambridge, Massachusetts, Alnylam Pharmaceuticals is at the forefront of transforming RNAi science into innovative medicines to improve patient outcomes.

Anthos Therapeutics, Inc. is a clinical-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on advancing therapies for cardiovascular and metabolic diseases. Founded in 2018, the company specializes in developing innovative treatments that target Factor XI and XIa, critical components of the intrinsic coagulation pathway. Its lead product, MAA868, is an anti-thrombotic investigational therapy designed to prevent thrombotic events while minimizing the risk of bleeding. By offering a long-acting treatment paradigm, Anthos Therapeutics aims to provide significant advantages over conventional standards of care, ultimately enhancing patient outcomes in cardiovascular health.

GRIN Therapeutics is a clinical-stage biotechnology company dedicated to developing precision therapeutics for serious pediatric neurodevelopmental disorders.