“We expect this collaboration will further validate our novel adeno-associated virus (AAV) gene therapy platform and support the development of new therapies that may allow for transformative treatments for these rare inherited eye diseases and other clinical indications”, Sue Washer, AGTC’s president and CEO, said in a statement. Biogen is also committed to helping fund AGTC’s R&D work, leaving the smaller biotech in charge of the lead program while taking the reins for the second gene therapy after AGTC wraps the first human study.
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Biogen will partner with AGTC to develop gene therapies for multiple eye diseases, in a collaboration that could generate more than $1 billion for AGTC. However, the news did not have much effect on Biogen’s shares and they continued to remain comparatively flat.
Both conditions, according to Biogen and AGTC, are also examples of unmet medical needs that may be addressed by replacing the single, faulty gene causing each disease.
Human trials are already conducted with treatments for X-linked Retinoschisis, a condition that fundamentally affects young males in their early years up to adolescence and can result in severe complications, such as retinal detachment or vitreous hemorrhage.
The forging of the deal showcased optimistic outlook with AGTC shares surging 21% to $19.75 in morning trading on the Nasdaq. Biogen will make additional payments, in accordance with fulfillment of several development and sales milestones agreed between the companies, which make the deal possibly worth more.
X-Linked Retinitis Pigmentosa, which commonly causes nyctalopia (night blindness) at the tender age of ten and elevates to legal blindness, is still in the pre-clinical phase. The transaction is expected to close in the third quarter of 2015.
Biogen, a giant in the field of multiple sclerosis treatment, said that under the deal, it would pay AGTC $124 million upfront and this includes a $30 million investment in the shares of AGTC. Genes control heredity and provide the basic biological code for determining a cell’s specific functions.
Gene therapy is still a new area.
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Through cutting-edge science and medicine, Biogen discovers, develops and delivers to patients worldwide innovative therapies for the treatment of neurodegenerative diseases, hematologic conditions and autoimmune disorders. Just last month, Avalanche Biotechnologies (NASDAQ:AAVL) stock plummeted on weak trial results for its gene therapy for a different eye disease, wet age-related macular degeneration.
