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Jun 30, 2014 bluebird bio Acquires Pregenen for Up to $139.9M
bluebird bio, a developer of gene therapies for severe genetic and orphan diseases, said today it acquired Precision Genome Engineering (Pregenen), in a deal that could have the buyer shelling out as much as $139.9 million for the company and its debt.
The deal gives bluebird access to Pregenen’s gene editing and cell signaling technologies, which the companies said could have a broad range of potential therapeutic applications in gene therapy as well as cancer immunotherapies.
Pregenen’s Homing Endonucleases (HEs), also called meganucleases, enable “multiplex” gene editing by targeting multiple genomic sequences for modification using a single gene delivery vector. HEs are designed to promote the editing or replacement of defective genes through the body’s natural cellular DNA repair mechanisms, by employing an integrated binding and cleavage mechanism to target DNA sequences greater than 20 base pairs in length. According to bluebird and Pregenen, HEs offer the potential for improved efficacy by unlocking multiple mechanisms of action within gene- and cell-based therapies.
Pregenen’s MegaTALs combine the natural DNA cleaving processes of HEs with the DNA binding region of transcription activator-like (TAL) effectors in a single-chain fusion enzyme. The companies say the protein fusion architecture of the MegaTALs enable the generation of highly specific and compact nucleases designed to be compatible with all current viral and nonviral cell delivery methods.
bluebird issued 408,667 shares of its common stock at the deal’s closing to former shareholders of privately held Pregenen, and agreed to pay or assume about $4.9 million of current liabilities of Pregenen and its shareholders.
The Pregenen shareholders are also eligible for up to an additional $15 million cash tied to achieving preclinical milestones, as well as $20.1 million cash for meeting clinical milestones and $99.9 million cash contingent on commercial milestones regarding product candidates identified using Pregenen’s technology.
“While we remain focused on driving our core programs forward, this acquisition represents a significant investment in our stated strategy to integrate emerging technologies that can enhance our ability to develop innovative and potentially transformative gene therapy and cancer immunotherapy products for patients,” Nick Leschly, bluebird’s president and CEO, said in a statement.
Added Pregenen co-founder Andrew Scharenberg, M.D.: “Teaming up with bluebird allows us to further expand and translate our gene editing and cell signaling technologies into potentially new and impactful human therapeutics, and provides an opportunity to complement bluebird’s established HSC and CAR-T cell product platforms to develop the next generation of gene therapy product candidates.”
Publicly traded bluebird is collaborating with Celgene in development of an early-stage CAR-T, or chimeric antigen receptor-modified T cell, program for oncology.
bluebird’s most advanced product candidate, Lenti-D, is in the recently launched Phase II/III Starbeam study assessing its effects on the treatment of childhood cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological disorder affecting young boys.
In addition, bluebird is in two Phase I/II studies for its next most advanced product candidate, LentiGlobin for beta-thalassemia major—the Northstar study in the U.S. and HGB-205 in France. HGB-205 also allows enrollment of one or more patients with sickle cell disease, with a separate U.S. sickle cell disease trial being planned, called HGB-206.
BLT Price at posting:
48.0¢ Sentiment: Hold Disclosure: Held
