Rare and orphan diseases often need generous outside investment or they will flounder.
If they get sufficient funding in the early days, they could be a wild success. If not...
Genzyme (now Sanofi) and others have been quite successful in the orphan disease market.
Many billionaires are wise investors, but many also put their money where they see the most need, rather than in the next biggest cash opportunity. -----
Any CAR-T immunotherapy that involves taking a patient's own immune cells and manipulating them to be effective against their specific disease is always going to be "bespoke" and very expensive, with no guarantee of success, although it may work wonders.
Any therapy that can be "off the shelf", meaning not having to be tailored quite so exquisitely to a particular patient, will be more cost effective, meaning more widely available and hence able to help more patients in the long run. Given the exploding cost of health care, even pre-Covid, this is a consideration.
At the end of Q2 20, the 13F filed by DE Shaw showed that the fund held 3940 positions (some positions are listed twice as long and also as call). PRTK is position #1622, ATRA is position #1751 and EIGR is position #2371.
They are not a significant holding in the fund so I would not pay any attention to it.