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Ophthotech Gets Rights to Gene Therapy for BEST Disease

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Ophthotech Corporation  announced that it has obtained development/commercialization rights to AAV gene-therapy program for BEST1-related retinal diseases from the University of Pennsylvania (Penn) and the University of Florida Research Foundation (UFRF)

In October, Ophthotech entered anexclusive option agreement with Penn and UFRF for rights to negotiate to acquire such a license. In the latest deal, the company converted this option and struck the exclusive global license agreement with the academic institutions

Shares of the company increased about 16% in after-hours trading, following the news. Shares of the company have rallied 20% compared with the industry’s growth of 9.5%.

Best disease, which generally affects individuals in both eyes, is an orphan inherited degenerative retinal disease. There is currently no FDA or EMA-approved therapy to treat this disease.

Based on current timelines and subject to regulatory review, Ophthotech expects to initiate a phase I/II study in the first half of 2021.

The deal is in sync with Ophthotech’s previously announced plans to evaluate promising gene-therapy product candidates in collaboration with leading companies and academic institutions.

Currently, the company has a series of sponsored research agreements with the University of Massachusetts Medical School, Penn and UFRF. These agreements allow the company to use the universities’ novel adeno-associated virus gene-therapy product candidates and gene-delivery methods targeting retinal diseases. Gene therapy is an emerging field, which can help develop transformational, next-generation therapies for treating ophthalmic diseases. In February 2018, Ophthotech initiated an innovative gene-therapy program to discover and develop new treatments for ocular disease.

Talking about gene-therapy programs for eye diseases, Spark Therapeutics, Inc. has a recently launched one-time gene-therapy product, Luxturna, in its portfolio. The drug is indicated for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy. It is currently marketed in the United States by Spark Therapeutics and elsewhere by Novartis (NVS - Free Report) . Notably, Spark Therapeutics has inked a $4.8-billion deal, per which it will be acquired by Swizz pharma giant Roche Holding AG (RHHBY - Free Report) .


Zacks Rank

Ophthotech currently carries a Zacks Rank #2 (Buy).  You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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