Roth Capital Partners has launched coverage of bluebird bio (NASDAQ:BLUE) with a “buy” rating and $50 price target. The stock closed at $36.63 on Friday.
“With its proprietary Lentiviral vectors, the company has successfully corrected dysfunctional genes and is well-positioned to deliver single treatment-based curative therapies for multiple monogenic diseases,” writes Debjit Chattopadhyay.
“Despite high transduction efficiency achieved in recent clinical studies, molecular clonality studies have not indicated increased risk of mutagenesis,” he added.
Mr. Chattopadhyay said that with the ability to deliver single-treatment cures for monogenic, chronic, fatal, genetic disorders, bluebird is challenging the current status quo. The current standard-of-care is not curative, excluding stem cell therapy, which is only available to less than 20% of patients, and is associated with significant mortality, morbidity, and economic burden as lifetime treatment costs exceed $500,000.
While gene therapy is unlikely to be cheap, curative treatments could deliver significant quality of life advantages for all patients, he added.
He said preliminary Phase 1/2 data in subjects with ß-thalassemia major is promising and data from the first two patients indicate transfusion independence within two weeks of therapy.
Lenti-D, bluebird’s most advanced and optimized gene therapy platform is being evaluated in patients with childhood cerebral adrenoleukodystrophy, a rare, hereditary neurological disorder that affects young boys and is often fatal. Prior studies have demonstrated the ability to halt disease progression in all four patients transplanted with Lenti-D, Mr. Chattopadhyay said.