AGTC XLRS Platform Well Tolerated

Early reports of AGTC’s lead gene therapy program for X-linked retinoschisis have shown the treatment to be well-tolerated with only mild-to-moderate inflammation that either resolves spontaneously or with steroids, AGTC president and CEO Susan B. Washer told the Posterior Segment Company Showcase at OIS@AAO 2016.

The XLRS platform is already in Phase I with eight patients enrolled and seven US centers participating, she said. AGTC is partnering with Biogen on the XLRS and XLRP platforms, the latter for X-linked retinitis pigmentosa. The next phase for the XLRP program is to file the IND, Washer noted.
But AGTC wholly owns rights to four other programs it’s developing, two each in acrhomatopsia and age-related macular degeneration (AMD). The AMD programs are still in the proof-of-concept phase, but the achromatopsia programs are further along: the CNGB3 program is in a dose-escalation Phase I/II trial, whereas CNGA3 is close to IND filing, Washer said.

She described AGTC’s potential treatments [OK?]as a “flexible platform” that can utilize any of the interdependent components of gene therapy to target specific genes. “We developed a very robust capability to design these vectors specifically for each and every indication that we’re working on,” Washer said. “In one, the capsid might be the most important; for another indication it might be the promoter; and for yet another indication it might be the formulation. But all these need to be designed together to specifically address the indication we’re working on.”

Washer also noted that AGTC holds $173 million in cash and expects to have at least $100 million in hand by next June, which should be sufficient to complete the XLRS and both achromatopsia Phase I/II trials and to initiate the Phase I/II XLRP trial and analyze data.

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