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In April 2015, AGTC and 4D Molecular Therapeutics, entered a collaboration and license agreement to discover and develop optimized AAV vectors to treat specific ophthalmic disease indications with high unmet medical need. 4DMT is a global leader in gene therapy product research & development using scientific innovation to unlock the full potential of gene therapy for patients with genetic conditions. As part of the agreement, 4DMT will deploy its proprietary AAV vector discovery platform, Directed Vector Evolution, to identify and optimize novel vectors in non-human primates. Directed Vector Evolution, empowers 4DMT to create customized gene delivery vehicles (novel AAV vectors) to deliver genes to any tissue or organ in the body.
In July 2015, AGTC and Biogen entered into a broad collaboration and license agreement to develop a portfolio of therapeutic candidates. The lead programs in the collaboration are X-linked Retinoschisis (XLRS) and X-Linked Retinitis Pigmentosa (XLRP). AGTC and Biogen will also collaborate on three additional jointly developed indications. In February 2016, the companies announced adrenoleukodystrophy (ALD) as the first of the three discovery programs under the collaboration. AGTC will lead the clinical development programs of XLRS through product approval and of XLRP through the completion of first-in-human trials. Biogen will also receive an exclusive license to use AGTC’s proprietary technology platform to make AAV vectors for up to six genes, three of which are at AGTC’s discretion, in exchange for payment of milestones and royalties.
In January 2017, AGTC entered into a strategic research and development collaboration with Bionic Sight, an innovator in the emerging field of optogenetics and retinal coding. Through this collaboration, AGTC and Bionic Sight will seek to develop a new optogenetic therapy that leverages AGTC’s deep experience in gene therapy and ophthalmology and Bionic Sight’s innovative neuro-prosthetic device and algorithm for retinal coding to develop new optogenetic therapies.
Dr. Sheila Nirenberg, CEO of Bionic Sight, explains her technology in these videos:
In June 2014, AGTC and SAFC signed an agreement to have SAFC provide gene therapy manufacturing services to companies developing gene therapy treatments using AGTC’s proprietary manufacturing methods. AGTC and SAFC will co-market AGTC’s proprietary manufacturing method with SAFC’s cGMP manufacturing capabilities. AGTC will assist clients in engineering AAV-based vectors and the associated helper viruses for manufacturing activities. SAFC will manufacture AAV vectors with the proprietary system to either support initial research or in accordance with cGMP requirements to support clinical trial development and eventual commercial production. Through this partnership, clients will also have access to bio-manufacturing testing services from BioReliance, SAFC’s biologics and early-development services business.
In December 2015, AGTC and Sympromics entered into a broad, multi-target collaboration agreement. Synpromics’ technology gives biological researchers, product developers and manufacturers control of gene expression through the ability to create a comprehensive portfolio of man-made DNA sequences. As part of the collaboration, the companies plan to utilize Synpromics’ proprietary technology to develop and optimize synthetic promoters for multiple cell types that will be used in the development of new gene therapy candidates.
AGTC has multiple Sponsored Research Agreements with the University of Florida to explore and develop novel technologies. These contracts cover basic research for many new treatment candidates as well as support clinical trials and research projects that will enhance the basic AAV technology platform. AGTC also holds multiple licenses from the University of Florida and has had a long-standing relationship with the university since the company’s founding.
Since 2016, AGTC has established multiple Sponsored Research Agreements to develop new therapies targeting inner ear diseases. AGTC’s research collaborations with Boston Children’s Hospital, Harvard Medical School, University of California San Francisco, and the Kresge Hearing Research Institute at The University of Michigan is expected to accelerate the translation of innovative approaches from the laboratory to the clinic, ultimately offering patients new treatment options for several inner ear diseases. The on-going research projects could advance several otology product candidates into clinical development in the next few years.