Gene Therapy progress with clinical trials, possible cures and major investment

Pfizer is establishing a gene therapy platform to study potential treatments, led by a top UK expert, and had struck a deal with privately owned U.S. biotech firm Spark Therapeutics to develop a treatment for haemophilia.

The Spark program is expected to enter early-stage clinical trials for haemophilia B in the first half of 2015. Spark will be responsible for the early Phase I/II tests, with Pfizer taking over late-stage studies, any regulatory approvals and potential commercialization.

Spark will get $20 million upfront and be eligible for additional payments based on product success worth up to $260 million.

Pfizer’s research effort in gene therapy will be led by Michael Linden, a professor from King’s College London and director of the University College London Gene Therapy Consortium. Linden is joining Pfizer on a two-year secondment.

Among other major pharmaceutical companies, Bayer AG struck a gene therapy deal with Dimension Therapeutics in June, while Novartis AG recently established a new cell and gene therapies unit, and Sanofi SA has a long-standing tie-up with Oxford BioMedica.

Bluebird Bio

Bluebird Bio has now treated seven beta-thalassemia patients with its experimental, one-time gene therapy. Four of the patients — all followed for longer than three months — are producing enough oxygen-carrying hemoglobin on their own to eliminate the need for chronic blood transfusions.

Two of these super-responding beta-thalassemia patients — followed for a year and nine months, respectively — have hemoglobin levels of healthy adults. At this point, a single infusion of Bluebird’s gene therapy has essentially cured them of this serious, inherited blood disease.

The remaining three beta-thalassemia patients were infused with Bluebird’s gene therapy around one month ago so it’s too early to assess their response. A single patient with sickle cell disease was also just treated within the past month.

Bluebird estimates there are about 15,000 patients with B-thal in the U.S. and Europe, a majority of which have the major genotype which requires regular blood transfusions and would be candidates for LentiGlobin.

In the ongoing “NORTHSTAR” study, five beta-thalassemia patients have been infused with LentiGlobin. The longest followed patient, at six months, is producing 3.8 g/dl of “marked” beta globin, a measure of functional hemoglobin produced by the working gene inserted by LentiGlobin. This represents 44% of the patient’s total 8.6 g/dl of hemoglobin, sufficient to be transfusion independent.

NOTE : I have investments in BLUE but not in the other mentioned stocks in this article

SOURCES – thestreet, reuters

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