Bluebird Bio, Inc. (BLUE) — our Top Pick for 2022 — is a clinical stage biotechnology company specializing in gene therapies for severe genetic disease and cancer, notes Joseph Cotton, editor of Cotton's Technically Speaking.
The company recently had two Biologics License Applications accepted for priority review by the FDA: 1) Beti-cel for b-thalassemia, and 2) Eli-cel for Cerebral Adrenoleukodystrophy.
The company also has a gene therapy candidate, lovo-cel, for sickle cell disease in a phase ½ clinical trial that has shown promising results, including stable production of gene therapy-derived anti-sickling hemoglobin and continued complete resolution of severe VOEs up to 36 months.
Patient-reported data on health-related quality of life (HRQoL) complemented clinical findings, and were the strongest HRQoL improvements in any sickle cell gene therapy program.
In December, the FDA placed its clinical program for lovo-cel gene therapy on partial clinical hold for patients under the age of 18. Enrollment and dosing for patients 18 and older in the clinical studies were not affected. For full disclosure, we have bought the stock and expect it to double within 6 months.