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Sana Biotechnology is focused on utilizing engineered cells as medicines for patients. The ability to modify genes and use cells as medicines will be one of the most important advances in healthcare over the next several decades. Sana is building differentiated capabilities across the spectrum of cell and gene therapy. Three aspirations drive Sana as we look to discover treatments for patients with poor outcomes or currently untreatable diseases. The first is the ability to repair and control the genes in any cell in the body. We are advancing novel delivery technologies with the goal of being able to deliver any payload to any cell in a specific, predictable, and repeatable manner, paving the way for next-generation in vivo gene therapy. Next is the ability to differentiate pluripotent stem cells ex vivo into immune-cloaked functional cells with the aspiration of being able to replace any missing or damaged cells in the body. Last is a belief we can enable broader access to our therapies through focusing on scalable manufacturing solutions, the cost of manufacturing, and aligning with key stakeholders. Sana launched in early 2019 and has approximately two hundred employees in Seattle, Cambridge, and South San Francisco. In 2019, we raised capital to enable our broad vision, began to acquire technology and establish an IP estate across cell and gene therapy, and made significant progress validating our cell and gene therapy platforms. Growing from this foundation, we intend to push forward our platforms and product pipeline in 2020 and beyond.
Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Our most advanced product candidate, apitegromab (SRK-015), a selective first-in-class inhibitor of the activation of the growth factor myostatin in skeletal muscle, is currently in development for the treatment of spinal muscular atrophy, or SMA. We are currently evaluating apitegromab in our TOPAZ Phase 2 proof-of-concept trial in patients with later-onset (Type 2 and Type 3) SMA. Positive six-month interim analysis results were announced in October 2020 and top-line data for the full 12-month treatment period are anticipated in the second quarter of 2021. In addition, we are conducting our DRAGON Phase 1 proof-of-concept trial for SRK-181, a highly specific inhibitor of latent TGFβ1 activation, in patients with locally advanced and metastatic solid tumors. We believe SRK-181 has the potential to overcome resistance and meaningfully increase the number of patients who may benefit from checkpoint inhibitors, such as anti-PD-1 or anti-PD-L1 therapies. Utilizing our proprietary platform, we are creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia.
RenovoRx is a clinical-stage biopharmaceutical company focused on fighting cancer through the localized treatment of difficult to treat tumors via our proprietary RenovoRx Trans-Arterial Micro-Perfusion (RenovoTAMP®) therapy platform. RenovoTAMP utilizes approved chemotherapeutics with validated mechanisms of action and well-established safety and side effect profiles, with the goal of increasing their efficacy, improving their safety, and widening their therapeutic window. Our lead product candidate, RenovoGem, is a combination of gemcitabine and our patented delivery system regulated by the FDA as a novel oncology drug product to treat unresectable locally advanced pancreatic cancer (LAPC). Targeted therapy via RenovoTAMP platform safely and without transmission to non-targeted areas, is our primary focus. RenovoRx`s patent portfolio currently includes seven issued US patents and one European patent for ourtechnology with several additional patents pending in the US, Europe, and Asia. After demonstrating a median survival of 27.9 months in Phase 1/2 clinical trials vs. 12-15 months reported in historical studies for this patient population, RenovoRx secured two separate Orphan Drug Designations from the FDA for intra-arterial gemcitabine: pancreatic cancer and bile duct cancer (cholangiocarcinoma). This therapy is being further studied in the currently enrolling Phase 3 TIGeR-PaC clinical trial in pancreatic cancer and has a primary endpoint of overall survival and several secondary endpoints, including quality of life. The Phase 3 TIGeR-PaC clinical trial is enrolling unresectable locally advanced pancreatic cancer patients (LAPC) in the United States.
Abnova Corporation is a Walnut, CA-based company in the Healthcare, Pharmaceuticals, and Biotech sector.
Founded in 2011, PANTHERx Rare is a specialty pharmacy solely focused on rare and orphan diseases, distributing several orphan products and providing access and support services to those needing them most. Known as The Rare Disease Pharmacy®, PANTHERx transforms lives by delivering therapeutic breakthroughs, clinical excellence, personalized support, and a range of access solutions to people living with rare and devastating conditions. While each rare condition affects few people, together all rare diseases impact an estimated 25 to 30 million Americans. Currently over 7,000 rare diseases have been identified and more than 90% of rare diseases are still without an FDA-approved treatment. Changes in federal policy and advances in science have led to a surge in FDA orphan drug approvals, providing tremendous hope to the rare disease community. PANTHERx is a dual-accredited specialty pharmacy, holding distinctions in rare disease including the Accreditation Commission for Health Care (ACHC) Distinction in Orphan Drugs and Utilization Review Accreditation Commission (URAC) Rare Disease Center of Excellence. As a pharmacy focused on patient satisfaction, PANTHERx is the seven-time winner of the prestigious MMIT Patient Choice Award, including the 2024 honor. PANTHERx is headquartered in Pittsburgh, Pennsylvania and is licensed in all 50 states and US territories. #ReinventingSpecialty #RevolutionizingPharmacy #RedefiningCare #DefiningRarePharmacy #TheRareDiseasePharmacy