BLUE - Is This Small-Cap Biotech a Buy After Its Second Approved Gene Therapy?
Despite recently getting its third drug approved, Bluebird Bio (NASDAQ: BLUE) only has a $1.7 billion market cap . Will the company overcome its past pricing issues and fly to new highs? And could its lead in sickle cell disease clinical trials translate into massive gains for investors?
Bluebird Bio already had a multiple myeloma CAR T-cell therapy, Abecma, approved earlier this year , and now it has not one, but two gene therapy approvals for rare genetic diseases in the EU. On July 21, the rapidly maturing biotech received EU approval for its latest gene therapy, Skysona, for cerebral adrenoleukodystrophy (CALD). This rare pediatric neurodegenerative disease affects boys, with just over 1,000 patients having been diagnosed in the U.S., and just over 6,000 in Europe. Devastating and irreversible, it worsens over time, causing deterioration of nerve cells in the brain. Most patients die within two years of diagnosis.
Before Skysona was approved, the only option available to CALD patients was a stem cell transplant -- a treatment that is less likely to cause complications if the donor is a sibling. However, it is estimated that fewer than 20% of CALD patients have a matched sibling donor.
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Is This Small-Cap Biotech a Buy After Its Second Approved Gene Therapy?