Gene therapy makers set to end 2024 in the red

BioMarin Pharmaceutical

31/12/2024 18:15

** Shares of bluebird bio  BLUE.O  leads yearly declines
for gene therapy makers, with Vertex Pharmaceuticals  VRTX.O 
and BioMarin  BMRN.O  also set to end 2024 in the red
      ** VRTX falls 1.6% YTD, while BMRN is down 32% and BLUE
down 71% 
    ** One-time treatments Lyfgenia and Casgevy, by BLUE and
VRTX, respectively, were approved in the U.S. last year to treat
inherited blood disorder sickle cell disease
     ** However, uptake of these gene therapies has been slow,
with early use seen mainly in patients with severe disease
    ** Centers for Medicare & Medicaid Services said this month
it had entered into agreements with VRTX and BLUE to help
increase patient access to their gene therapies
    ** BLUE's shares fell to record low of 32 cents in November
after co missed third-quarter revenue estimates 
    ** BMRN said in August it will refocus commercial operations
for hemophilia gene therapy Roctavian on three key markets; said
it will reduce its global workforce by about 225 employees 
    ** YTD, VRTX down ~2% vs a 40% gain in 2023, BMRN set for
its second straight year of decline and BLUE set for its fifth
straight year of decline

 (Reporting by Sneha S K)
 ((Sneha.SK@thomsonreuters.com;))