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Vor Biopharma is pioneering engineered hematopoietic stem cells (eHSCs) to dramatically change the treatment paradigm for hematological cancers. Vor`s eHSCs are designed to generate healthy, fully functional cells with specific advantageous modifications, for example to protect the blood and bone marrow from the toxic effects of antigen-targeted therapies, protecting healthy cells from depletion but leaving tumor cells vulnerable. Vor`s platform could potentially be used to change the treatment paradigm of both hematopoietic stem cell transplants and antigen-targeted therapies such as antibody drug conjugates, bispecific antibodies and CAR-T cell treatments. A proof-of-concept study for Vor`s lead program has been published in Proceedings of the National Academy of Sciences. Vor is based in Cambridge, Mass. and has a broad intellectual property base including in-licenses from Columbia University, where foundational work was conducted by inventor and Vor Scientific Board Chair Siddhartha Mukherjee, MD, DPhil. Vor was founded by Dr. Mukherjee and PureTech Health and is supported by leading investors including 5AM Ventures and RA Capital Management, Johnson & Johnson Innovation – JJDC, Inc. (JJDC), Novartis Institutes for BioMedical Research and Osage University Partners.
Marina Biotech`s focus is to treat the intersection of arthritis, pain, hypertension, and oncology diseases using combination therapies of already approved drugs. The company is developing and commercializing late stage, non-addictive pain therapeutics. The company`s `next-generation of celecoxib,` including IT-102 and IT-103, are designed to control the dangerous side-effect of edema that prohibits the drug from being prescribed at higher doses. These have the potential of replacing opioids and combating the opioid epidemic. They are also being developed as anticancer agents against FAP and CRC. On the Sales and Marketing front- we are actively promoting sales and distribution of our FDA approved drug- Prestalia- through our patient centric care program: 1) We have developed a patented total care program which increase compliance to over 80%. 2) Increase compliance is expected to improve care for hypertensive patients, especially resistant hypertensive patients which currently have no treatment available to them. 3) Resistant hypertension market size is large with 22% of all hypertensive pts eventually become resistant hypertension 4) Compliance/counseling has been shown clinically to treated resistant hypertensive patients 5) Deep pipeline a. Perindopril- Hypertension monotherapy. ACEON (Perindopril) NDA b. Prestalia- Hypertension combination therapy. i. Perindopril/Amlodipine, ii. 1st line hypertension, c. Atorvastatin/Perindopril: Hypertension and lipid disorder d. Pipeline drug: Hypertension and Pain. IT-102, IT-103
Cytokinetics is a late-stage biopharmaceutical company focused on discovering, developing and commercializing first-in-class muscle activators as potential treatments for debilitating diseases in which muscle performance is compromised and/or declining. As a leader in muscle biology and the mechanics of muscle performance, the company is developing small molecule drug candidates specifically engineered to increase muscle function and contractility. Cytokinetics’ lead drug candidate is tirasemtiv, a fast skeletal muscle troponin activator, for the potential treatment of ALS. Tirasemtiv has been granted orphan drug designation and fast track status by the U.S. Food and Drug Administration and orphan medicinal product designation by the European Medicines Agency for the potential treatment of ALS. Cytokinetics retains the right to develop and commercialize tirasemtiv. Cytokinetics is collaborating with Amgen Inc. to develop omecamtiv mecarbil, a novel cardiac muscle activator, for the potential treatment of heart failure. Cytokinetics is collaborating with Astellas Pharma Inc. to develop CK-2127107, a fast skeletal muscle activator, for the potential treatment of spinal muscular atrophy and chronic obstructive pulmonary disease. Amgen holds an exclusive license worldwide to develop and commercialize omecamtiv mecarbil and Astellas holds an exclusive license worldwide to develop and commercialize CK-2127107. Both licenses are subject to Cytokinetics` specified development and commercialization participation rights.
AVROBIO`s vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease. AVROBIO is powered by our industry leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. We are headquartered in Cambridge, Mass., with an office in Toronto, Ontario.
Excision BioTherapeutics is a private company focused on developing advanced gene editing therapeutics to address the medical need for the treatment of life-threatening disease caused by viral infections. Our mission is to advance gene editing therapeutics for our indications into safe and efficacious medicines that will greatly improve the lives of infected individuals around the world.