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Cydan Development, Inc. 700 Technology Square, 3rd Floor Cambridge MA 02139 USA +1 617 231-6020 [email protected] www.cydanco.com Fact Sheet Accelerating Rare Genetic Disease Therapies About Cydan The Cydan Team Cydan Development is the first orphan drug accelerator dedicated to advancing therapies that improve the lives of people living with rare genetic diseases. Cydan starts with a compelling scientific idea or promising drug candidate from collaborators in academia CHRIS ADAMS, PhD, MBA Founder and Chief Executive Officer and industry, and then efficiently advances therapies through pre-clinical R&D to enable the establishment of an independent company to accelerate the therapy through clinical development and ultimately to the people who need them. JAMES McARTHUR, PhD The accelerator was launched in 2013 with a collaborative team of Founder and Chief Scientific Officer scientists and entrepreneurs with comprehensive experience and expertise in drug discovery, clinical development and business development. In early 2015, we launched our first new company – Vtesse – which is developing drugs for Niemann-Pick Disease Type C (NPC) and other rare, severe diseases with great unmet need. VERED BISKER-LEIB, PhD, MBA Chief Business Officer Access to Capital Cydan works with leading investors in the life sciences space. The accelerator was funded with $26 million in a financing led by New Enterprise Associates (NEA), Pfizer Venture Investments, AILEEN HEALY, PhD Vice President, Preclinical Development Lundbeckfond Ventures, Bay City Capital, and Alexandria Real Estate Equities, Inc./Alexandria Venture Investments. Additional capital is available from the investor syndicate to launch the NewCos. Cydan Newcos Vtesse, which Cydan launched in January 2015, is developing DIONE KOBAYASHI, PhD Vice President, Preclinical Translation VTS-270, a drug for patients with Niemann-Pick Disease Type C (NPC) and other rare, severe diseases with great unmet need. Vtesse was started with an initial Series A round of funding, an experienced management team, a compelling corporate story and as well as a path to human clinical proof of concept. For LAURA ALESSIO more information, visit www.vtessepharma.com. Office Manager Cydan Fact Sheet Opportunities Opportunities Launch New Companies THE CYDAN MODEL Screen/Diligence Our Rare Disease Drug Development Model We scour scientific literature, visit universities and attend medical conferences to identify potential compelling therapies. Our well-financed, transparent, four-part model is unique and truly Screen/Diligence Our rapid assessment internal scenarios. and external We build the development plan adaptable towith multiple resources determines the path forward for this collaboratively, new clinical candidate. share all the data from our studies and accelerate decision-making. De-risking An IND-ready asset is ready for Series A funding with a NewCo to bring this orphan drug candidate Our goal is the same as yours: to rapidly to patients. advance development of therapies that will impact the lives of people with living with Launch New Companies rare genetic diseases. Cydan NewCos have an investible corporate story, access to capital, experienced management and a clear path forward. De-risking Opportunities The Cydan team researches the scientific literature, visits universities, and attends medical conferences to identify compelling therapies that have the potential to significantly impact monogenic rare diseases and change lives. Cydan opportunities have arisen from collaborations with: • Academic laboratories • Government-funded labs • Small biotech/biopharma companies • Large pharma R&D Diligence and Screening Cydan conducts a rapid assessment to determine a path forward. Our screening process seeks answers to these questions: • Is there an asset or a path to this asset? • Is the therapy targeting the disease pathway or a significant symptomatology? • Is this a disease with significant unmet need? • Is the potential patient population sufficient for clinical development and commercialization? • Is there intellectual property (IP) or a path to IP protection for the asset? During diligence, Cydan identifies and develops a path forward with extensive internal and external resources, including experts in: • Disease Pathology and Epidemiology • Manufacturing • Toxicology • Clinical Development and Regulatory • Chemistry • Intellectual property During diligence, we seek to gain a full appreciation of the mechanism of action behind potential therapies and determine if this mechanism of action will fundamentally change the pathophysiology of the underlying disease. At this stage we may decide the scientific idea is compelling but we must create a new clinical candidate. De-Risking De-risking yields an IND-ready asset considered ready for company launch to bring this orphan drug candidate to people living with a rare genetic disease. We conduct a thorough analysis to: • Expand pharmacology with in vitro and in vivo studies • Determine a viable manufacturing strategy • Address any toxicology risks • Develop the IP strategy • Establish the disease epidemiology, commercial market opportunity and the competitive landscape • Construct a clinical and regulatory development pathway NewCo Launch Cydan brings more to company creation than access to our VC network. We bring an experienced team to lead successful company formation including: • Creation of a compelling corporate story • Manage the investor diligence and Series A raise • Identify the management team • Ongoing NewCo guidance and support Cydan Development, Inc. 700 Technology Square, 3rd Floor Cambridge MA 02139 USA +1 617 231-6020 [email protected] www.cydanco.com