Some US gene therapy developers set to end 2023 on a high

Crispr Therapeutics AG

29/12/2023 16:46

** Shares of some U.S.-listed gene therapy makers on track
to end the year higher with Vertex Pharmaceuticals  VRTX.O  and
Krystal Biotech  KRYS.O  rising about 41% and 58%, respectively
    ** Both the companies received U.S. regulatory approvals in
the year - KRYS' Vyjuvek for treating a rare skin-disorder and
VRTX's Casgevy for sickle cell disease
    ** Gene therapy companies will remain in focus of business
development and M&A discussions in 2024 as larger drugmakers
look to freshen up their pipelines - brokerage Leerink Partners
    ** Gene therapies are at a lower risk of exposure to price
renegotiations under U.S. Inflation Reduction Act - brokerage
    ** Sarepta Therapeutics  SRPT.O  fell about 24% YTD vs rise
of 44% in 2022; company received accelerated approval for
Elevidys in June 2023 to treat a rare muscular dystrophy, but
for a smaller patient population than the company was seeking
    ** bluebird bio  BLUE.O  stock down 79.7% YTD on investor
concerns over higher price and serious safety risk for its
recently approved sickle cell disease therapy Lyfgenia
    ** BioMarin Pharmaceutical  BMRN.O  fell about 6% in 2023,
snapping its three-year winning streak; FDA greenlit its gene
therapy Roctavian to treat an inherited bleeding disorder 
    ** KRYS on track for its sixth straight year of gains, while
BLUE is on pace for its sixth straight year of losses

 (Reporting by Pratik Jain in Bengaluru)
 ((Pratik.Jain@thomsonreuters.com))