Bioblast Pharma Ltd. (Nasdaq:ORPN), a clinical-stage, orphan disease-focused biotechnology company, today announced results of a six-month open label Phase 2a study that also included an additional six-month follow-up period investigating trehalose in patients with Spinocerebellar Ataxia Type 3 (SCA3).The objectives of the study were to establish safety and tolerability of
Global Blood Therapeutics (NASDAQ:GBT) today announced enrollment of the first patient in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a pivotal Phase 3 clinical trial of GBT440 in people with sickle cell disease (SCD). The HOPE Study will be conducted at leading SCD sites globally and
Pharmaceutical companies have developed numerous strategies to counter the patent cliff. One such tactic? Capitalizing on the personalized medicine market. As it turns out, this niche market is less vulnerable to the patent cliff—which may make it attractive to pharmaceutical companies and investors alike.
The past ten years have seen pharmaceutical companies take a different approach to developing respiratory drugs, making them more targeted to specific diseases.
While there has still yet to be a single gene therapy approved in the United States, Europe is now closer to marketing another: bluebird bio was just granted accelerated assessment status for LentiGlobin, a gene therapy that targets sickle cell disease.
GenSight Biologics, a France-based biotech company, is developing a new gene therapy specifically for a debilitating eye disease condition. Currently in phase III trials, the treatment is designed to both prevent and restore vision loss in patients.
By Vivien Diniz
Amicus Therapeutics (NASDAQ:FOLD), a biotechnology company at the forefront of rare and orphan diseases, today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion to approve the oral small molecule pharmacological chaperone migalastat as a first line therapy for Fabry disease in
AstraZeneca’s (NYSE:AZN) drug olaparib appears promising for a targeted group of prostate cancer patients, united by a distinct genetic mutation.
The company’s announcement points to a larger trend towards biotech innovation in the San Diego area.
Today’s technologies give parents more options for discovering and in some cases potentially preventing genetic abnormalities. Companies that can uncover new methods for genetic testing and acquire new technology investment may be in a good position for future growth.