Gene therapy focus: Pfizer buys Bamboo, Shire axes Baxalta programme

Pfizer this week announced it has bought North Carolina-based private biotech firm Bamboo Therapeutics for an upfront payment of $150m (€135m).

The deal adds an advanced recombinant Adeno-Associated Virus (rAAV) vector design and production technology, and several pipeline therapies including a Phase I candidate for Giant Axonal Neuropathy, and a preclinical programmes targeting Duchenne Muscular Dystrophy (DMD).

Gene therapy is the introduction of normal genes into cells in place of missing or defective ones using a technology such as a viral vector in order to correct a patient’s genetic disorders.

“We believe Bamboo’s industry leading capabilities in rAAV vector design and manufacturing complement Pfizer’s rare disease strategy,”​ Gregory LaRosa, CSO of Pfizer’s rare diseases unit said in a statement.

Mikael Dolsten, president of Pfizer Worldwide R&D added the deal further enhances the firm’s “leadership position in this area.”​

He continued: “Gene therapy may hold the promise of bringing true disease modification for patients suffering from devastating diseases, and we hope to see this promise come to fruition – through new and existing in-house capabilities and potential partnership opportunities – in the years to come.”​

Pfizer also gains an 11,000sq ft Phase I/II gene therapy manufacturing facility in North Carolina - bought by Bamboo earlier this year from the University of North Carolina at Chapel Hill – which is based on a suspension cell based production platform that the firm says offers scalability, efficiency and purity due to the use of serum free and chemically defined media.

Shire halts Baxalta programme​

In other gene therapy news, Shire - fresh from its $32bn merger - has decided to scrap development of Baxalta’s factor IX gene therapy candidate Bax 335, which had been in Phase I/II trials for haemophilia B.

“The expression was good but it was a little inconsistent between different patients,”​ said Shire’s head of R&D Philip Vickers in a conference call discussing Q2 earnings this week. “With time, for some patients, the level of expression decreased. And we think that's a very important thing to factor in when considering all gene therapies, is the expression going to go down over time.”​

Instead Shire will pursue its own “next-generation preclinical programme,”​ committed to gene therapy within haemophilia, and has its own haemophilia A gene therapy programme looking to enter clinical studies later this year.

“We've been talking now for some time about our growing interest in gene therapy and wanting to get access to the right vectors and experience in manufacturing,”​ said Vickers.

While acknowledging the firm is working on a “classic gene therapy where you'll get a protein and try and express it,” ​he added there are aspects of gene editing as well as just gene replacement that are of interest to Shire.