Gene Therapy Briefs

Avalanche Biotechnologies. Avalanche Biotechnologies, Inc. announces positive top-line phase 2a results for AVA-101 in wet age-related macular degeneration. Available at: http://investors.avalanchebiotech.com/phoenix.zhtml?c=253634&p=irol-newsArticle&ID=2059444 (accessed August 31, 2015).

Garde D. Avalanche tanks after its gene therapy posts mixed results in AMD. Available at: www.fiercebiotech.com/story/avalanche-tanks-after-its-gene-therapy-posts-modest-effect-amd/2015-06-16 (accessed August 31, 2015).

Biogen and AGTC. Biogen and AGTC enter collaboration to develop gene therapies in ophthalmology. Available at: http://media.biogen.com/press-release/investor-relations/biogen-and-agtc-enter-collaboration-develop-gene-therapies-ophthalm (accessed August 31, 2015).

Biogen. Form 10-Q, filed 07/24/15 for the period ending 06/30/15. Available at: http://api40.10kwizard.com/cgi/convert/pdf/BIIB-20150724-10Q-20150630.pdf?ipage=10395297&xml=1&quest=1&rid=23&section=1&sequence=-1&pdf=1&dn=1 (accessed August 31, 2015).

Applied Genetic Technologies Corporation (2015). AGTC Announces Achievement of XLRS Patient Enrollment Milestone in Biogen Collaboration. Available at: http://ir.agtc.com/releasedetail.cfm?ReleaseID=929084 (accessed September 2, 2015).

Adaptimmune. Adaptimmune's NY-ESO-1 TCR-engineered T-cells demonstrate durable persistence, clinical activity and tolerability in clinical study in multiple myeloma patients. Available at: www.adaptimmune.com/wp-content/uploads/2015/07/ADAP-Nature-Medicine-release-20-July-2015.pdf (accessed August 31, 2015).

Baxalta. Baxalta launches as a global biopharmaceutical leader dedicated to patients with orphan diseases and underserved conditions. Available at: www.baxalta.com/newsroom/press-releases/baxalta-day-one.page? (accessed August 31, 2015).

Baxalta. Baxalta reports continued progress on phase 1/2 clinical trial of BAX335, investigational gene therapy treatment for hemophilia B. Available at: www.baxalta.com/newsroom/press-releases/clinical-trial-bax335.page? (accessed August 31, 2015).

Baxalta. Baxalta completes ONCASPAR portfolio acquisition, aims to broaden patient access to leading treatment for leukemia. Available at: www.businesswire.com/news/home/20150723005650/en/Baxalta-Completes-ONCASPAR-Portfolio-Acquisition-Aims-Broaden#.VcTPB_mJThk (accessed August 31, 2015).

Celgene and bluebird bio. Celgene and bluebird bio to develop anti-BCMA product candidates; bluebird bio regains rights to CAR T programs outside of BCMA. Available at: http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2056226 (accessed August 31, 2015).

Kite Pharma and bluebird bio. Kite Pharma and bluebird bio announce strategic collaboration to advance second generation TCR cell therapy products to treat HPV-associated cancers. Available at: http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2061313 (accessed August 31, 2015).

bluebird bio and Five Prime Therapeutics. bluebird bio and Five Prime Therapeutics enter into license agreement for novel antibodies to develop chimeric antigen receptor T cell therapy. Available at: http://investor.bluebirdbio.com/phoenix.zhtml?c=251820&p=irol-newsArticle&ID=2053589 (accessed August 31, 2015).

Celladon. Celladon Corporation provides business update. Available at: http://ir.celladon.com/releasedetail.cfm?ReleaseID=919606 (accessed August 31, 2015).

Celladon. Form 8-K, filed 05/14/15 for the period ending 05/14/15. Available at: http://files.shareholder.com/downloads/AMDA-27TNHK/0x0xS1193125-15-188141/1305253/filing.pdf (accessed August 31, 2015).

Celladon (2015). Celladon Reports Second Quarter 2015 Financial Results. Available at: http://ir.celladon.com/releasedetail.cfm?ReleaseID=927195 (accessed September 2, 2015)

Celladon (2015) Form 10-Q Quarterly Report, filed with the U.S. Securities and Exchange Commission. Available at: http://files.shareholder.com/downloads/AMDA-27TNHK/659788777x0xS1193125-15-286510/1305253/filing.pdf (accessed september 2, 2015)

Celgene and Juno Therapeutics. Celgene and Juno announce ten-year collaboration to advance potentially groundbreaking immunotherapies for patients with cancer and autoimmune diseases. Available at: http://ir.celgene.com/releasedetail.cfm?releaseid=919929 (accessed August 31, 2015).

Juno Therapeutics. Juno announces early HSR clearance for global collaboration with Celgene. Available at: http://ir.junotherapeutics.com/phoenix.zhtml?c=253828&p=irol-newsArticle&ID=2075973 (accessed August 31, 2015).

U.S. Food and Drug Administration. Considerations for the design of early-phase clinical trials of cellular and gene therapy products: guidance for industry. Available at: www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM359073.pdf (accessed August 31, 2015).