UPDATED: Celgene makes big splash in gene therapy with two key cancer collaborations

Celgene has made a move into the resurgent gene therapy field. With a focus on immunotherapies against cancer, Celgene revealed collaborations with a pair of partners on Thursday that highlight increased industry interest in the previously shunned gene therapy field after a series of regulatory and scientific victories in recent years.

The biopharma powerhouse ($CELG) has joined the ranks of Novartis ($NVS) and other industry players to invest in research of new gene therapies that use modified immune cells from a patient to attack his cancer. In small trials involving patients with forms of blood cancers, researchers from the University of Pennsylvania and a separate group at Memorial Sloan-Kettering Cancer Center have shown how such treatments rapidly wiped out tumor cells.

Celgene has latched onto expertise in so-called chimeric antigen receptor (CAR) T-cell therapies from Baylor College of Medicine's cell and gene therapy center and affiliated hospitals in Texas. The Summit, NJ-based biopharma company, which sells the blockbuster cancer drug Revlimid, plans to collaborate with Baylor's Dr. Malcolm Brenner and Cambridge, MA-based bluebird bio (a 2012 Fierce 15 company) to advance research of gene therapies in oncology.

"The genetic manipulation of autologous T-cells is a new frontier in oncology, one that shows early promise in emerging clinical trials," said Tom Daniel, Celgene's president of research and early development, in a statement.

Bluebird, a gene therapy specialist that raised a $60 million venture round in July, says that the company would handle research and clinical development of any therapies from its Celgene collaboration through Phase I trials. Celgene has forked over an undisclosed upfront sum and agreed to pay out up to $225 million in option fees and milestones to bluebird for each therapy that results from the research.

Nick Leschly, CEO of bluebird bio

Gene therapy has begun to inspire new interest after past attempts to alter the genetics of cells to treat disease ended in disaster. After years of struggle, the Dutch biotech uniQure last year grabbed the first approval of a gene therapy in the West with European regulators sanctioning the company's treatment for lipoprotein lipase deficiency. And bluebird aims to be among the next commercial gene therapy outfits with its lead clinical-stage program in childhood cerebral adrenoleukodystrophy (ALD), which the company plans to push into a Phase II/III trial this year.

While Celgene brings expertise in cancer drug development and Baylor in CAR T-cell biology, bluebird CEO Nick Leschly told FierceBiotech, his biotech has experience using lentiviral vectors to deliver genes into target cells taken from a patient's body. Its method involves first extracting the cells from a patient, delivering desired genes into his cells with its viral vectors, and then giving the modified cells back to him to treat his disease. The collaborators plan to research CAR T-cell therapies for liquid cancers as well as solid tumors.

The group's lentiviral therapy proved effective in two patients with ALD, a rare neurological disorder, whose disease was stabilized for more than four years after treatment as of June 2012. 

"I think it comes down to the work that we've built in our platform and what we're bringing to the table in ex-vivo lentiviral therapy, whether it's intellectual property, whether it's knowhow," Leschly said of the new collaboration. Bluebird also brings "the people on the ground who are executing this, which is what you need to do in gene therapy and specifically with CAR T-cells."

- here's the release

Special Report: Bluebird Bio – 2012 Fierce 15