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bluebird (BLUE) Submits BLA for Gene-Therapy to Treat Thalassemia

bluebird bio, Inc. BLUE announced that it has completed the rolling submission of its biologics license application (“BLA”) to the FDA for betibeglogene autotemcel (“beti-cel”) for the treatment of all patients with transfusion-dependent β-thalassemia (“TDT”) across all ages and genotypes.

TDT is a genetic disorder caused by mutations in the β-globin gene that results in adult hemoglobin (“Hb”) reduction, chronic anemia and lifelong dependence on transfusion of red blood cells.

In the year so far, bluebird’s stock price has declined 58.4% in comparison with the industry’s 0.6% decline.

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The BLA submission is based on data from multiple clinical studies, the phase III HGB-207 (Northstar-2) and HGB-212 (Northstar-3) studies, and the phase I/II HGB-204 (Northstar) and HGB-205 studies, which evaluated beti-cel in 63 patients of all ages across β°/β° and non-β°/β° genotypes (as of Mar 9, 2021). All the studies, on a combined basis, represent more than 220 patient-years of experience with beti-cel.

The company is also conducting a long-term follow-up study, LTF-303, to monitor the safety and efficacy of people who have participated in beti-cel clinical studies.

The FDA has already granted Orphan Drug and Breakthrough Therapy designations to beti-cel for TDT indication.

Once approved, beti-cell will be the first hematopoietic stem cell (HSC) ex-vivo gene therapy for patients in the country.

In June 2019, the therapy received conditional marketing authorization in the European Union (EU) for a similar indication and is marketed under the trade name Zynteglo, making it the first gene therapy approved for this indication in the EU.

Apart from beti-cel, the company is also developing a CART-T therapy bb21217 (for multiple myeloma). The company also has partnerships with bigwigs like Bristol Myers Squibb BMY and Regeneron REGN to boost its pipeline development.

Zacks Rank & Stock to Consider

bluebird currently carries a Zacks Rank #3 (Hold). A better-ranked stock in the same sector is Repligen Corporation RGEN, which currently holds a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Repligen’s earnings per share estimates for 2021 have increased from $2.26 to $2.76 in the past 60 days. The same for 2022 has risen from $2.57 to $3.03 over the same period. The stock has rallied 64% in the year so far.

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