Replies to post #193236 on Biotech Values

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AGTC > The Market Is Lukewarm Towards Applied Genetic's New Partnership With Biogen For Good Reason

http://seekingalpha.com/article/3322425-the-market-is-lukewarm-towards-applied-genetics-new-partnership-with-biogen-for-good-reason

This then is what concerns me and may be the reason for the market's lukewarm reaction to the Biogen deal and the downward trend in AGTC stock price since the end of January:

rAAV-based gene therapy programs abound.
AAVL has a lead in gene therapy directed at wet AMD for which AGTC is gearing up; AGTC will also be competing with its former partner Genzyme.
AAVL will compete with AGTC for the small, orphan XLRS market.
rAAV may not be the vector of choice even for diseases of the eye.
The disappointing results of gene therapy for the Leber RPE65 mutation, cited above, are discussed in an editorial by Dr. Wright here in the same May 15 issue of NEJM. He postulates that gene therapy might have resulted in sustained improvement if it had been started at an earlier stage of the disease (the same has been said about gene therapy for Duchenne muscular dystrophy). He also suggests that a more efficient rAAV vector or lentiviral vector may be needed. Sue Washer, AGTC CEO, offered a much more optimistic view of the Leber study results and implications for other eye diseases at the Cantor Fitzgerald Healthcare conference posted here.

There are a lot of scientists in the industry and in academia targeting single-gene diseases and using rAAV platforms. Successful transduction with a vector does not ensure durable clinical results. Repeat dosing may be met with resistance from the immune system (T-cells). Long-term success could still be a long way off for AGTC, for its new partner in gene therapy, and for its competitors. I admire AGTC which in many ways is just what I look for in an investment, but at $0.3B, this tiny company seems reasonably priced. I share the market's reluctance to bid the price up much on the Biogen news.