bluebird bio (blue 4.73%) When CRISPR Therapeutics (CRSP 4.07%) Two start-up biotech companies specializing in gene-editing therapies to treat rare inherited blood disorders and cancer.
Bluebird’s stock is down more than 16% over the past year and is up more than 8% over the past three months. CRISPR’s stock has fallen more than 27% over the past year and more than 8% over the past three months, but the fortunes of both stocks could change dramatically this year.
Both companies are far from profitable, each making about $100,000 in profit last quarter, all from collaboration revenue. However, both have attractive pipelines that could pay off quickly. Which of these long-term investments is the better buy? Let’s find out.
Bluebird Bio case
Bluebird Bio’s biggest advantage over CRISPR Therapeutics is that there are already two gene-editing therapies approved by the Food and Drug Administration (FDA). Meanwhile, CRISPR is still awaiting its first FDA-approved therapy.
Bluebird’s Zynteglo, a one-time curative therapy for the rare genetic blood disorder transfusion-dependent beta-thalassemia (TDT), was approved by the FDA in August and is already in use in at least one commercial patient (This therapy is priced at $2.8 million). , the company said earlier this month that it expects revenue from the drug to begin in the first quarter of 2023.
The Company’s other approved therapy, Skysona, received FDA approval in September to treat cerebral adrenoleukodystrophy (CALD). Priced at $3 million he is the most expensive drug in the world.
About 5,000 people in the United States have beta thalassemia, according to the National Center for Translational Sciences. CALD only affects her 1 boy in 20,000, according to the Child Neurology Foundation. Such a small market further limits sales due to the company’s high cost of treatment, but at that price Bluebird doesn’t need many patients to be profitable.
The company also has another late-stage therapy, lovo-cel, to treat sickle cell disease (SCD), and the company plans to submit a biologics license application (BLA) to the FDA. said it is. First quarter of 2023.
The Case of CRISPR Therapeutics
Gene therapy is expensive to get started, and CRISPR has a much bigger force than Bluebird Bio. As of Sept. 30, the company had $1.973 billion in cash, but Bluebird said that even after selling Priority Review vouchers for $200 million, it had only about $382 million in cash, and in December Added $182 million listed as of 31st. That’s enough. , Bluebird said it will last until the first quarter of 2024.
The lead therapy for CRISPR is exa-cel in development. Vertex PharmaceuticalsIf approved by the FDA this year, exa-cel could have an edge over Zynteglo. Because its price is likely to be less than $2.8 million, it appeared marginally effective in trials. ExaCel, in clinical trials, was able to eliminate her need for transfusion in 42 of her 44 TDT patients, and reduced her need for transfusion in 2 of her other patients from 75% to 75%. He has decreased by 85%. It also stopped painful vascular occlusive crises in all 21 patients with sickle cell disease. Zynteglo was effective in curing him 90% of TDT patients. Still effective, but not as effective as Exacel, Zynteglo is not approved for SCD.
CRISPR only takes 40% of the revenue from exa-cel, while Vertex takes the rest, but the potential for this drug is huge. Both companies have already filed for approval in Europe and the UK, and his BLA filing in the US is phasing and is expected to be completed by the end of the first quarter of 2023.
CRISPR also has a larger and more diverse pipeline than Bluebird, with CTX110, a donor-derived CAR T-cell gene therapy that shows promise in treating refractory and relapsed large B-cell lymphoma in a single dose. It is shown that there is The company is also eyeing CTX130, an allogeneic CAR T-cell gene therapy, as well as CTX110. As a potential blockbuster, CTX130 has performed well in early trials to treat relapsed or refractory T-cell or B-cell malignancies.
Bluebird Bio is off to a head start on approvals, but we believe CRISPR has more long-term potential due to exa-cel’s effectiveness, the company’s strong cash position and larger pipeline. . CRISPR is mentioned more often as a buyout candidate, which will of course reward current shareholders.
James Halley holds a position at CRISPR Therapeutics. The Motley Fool US headquarters holds positions with and recommends CRISPR Therapeutics and his Vertex Pharmaceuticals. The Motley Fool recommends Bluebird his bio stock. The Motley Fool’s U.S. headquarters has a disclosure policy.