AGTC Moves Past Biogen With Ophthalmic Gene Therapy

 
• By Joseph Haas
  
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AGTC hopes to gain the first approval in X-linked retinitis pigmentosa for its gene therapy AGTC-501, now in a Phase II/III study. It also is advancing development in achromatopsia, another unmet medical need.

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AGTC is advancing two gene therapies for rare retinal disorders

Applied Genetic Technologies Corporation has emerged as presumptive leader in bringing the first drug therapy for X-linked retinitis pigmentosa (XLRP) to market in the US and announced plans to advance another gene therapy into Phase II in a specified population of achromatopsia patients, also an area in need of new treatments. This led one analyst to describe AGTC as “more than meets the eye” with its focus on adeno-associated vector (AAV)-delivered gene therapies for rare ophthalmic disorders.

Gene therapy is still a risky field, but Gainesville, FL-based AGTC execs used the firm’s 22 July investor day to...

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