Martin Shkreli posted this on his blog in August:
"The next five or ten years of biopharma investing is basically all about gene therapy. After AVXS, who knows what is possible. Abeona (ABEO), Axovant (AXON), Audentes (BOLD), BioMarin (BMRN), Oxford, PTC (PTCT), Spark (ONCE), UniQure (QURE), Rocket (RCKT), Sangamo (SGMO), RGNX, Sarepta (SRPT), Solid (SLDB), Ultragenyx (RARE), Voyager (VYGR) are all players. My guess is an equal-weighted or capitalization-weighted index of the above would outperform pretty much any other index. "
Spark just got bought for a 120% premium.
He's also very bullish on GBT.
I'd have to look deeper into it but there's some compelling names there.