Acquisition brings Homing Endonuclease and MegaTAL gene-editing and novel cell signaling technologies with a broad range of potential therapeutic applications in gene therapy and cancer immunotherapies
“We are excited to add Pregenen’s team and their unique gene editing
platform to bluebird as it will further expand and lever our gene
therapy product platform,” stated
“Pregenen is a recognized leader in the development and reprogramming of
novel Homing Endonuclease and MegaTAL based enzymes that provide a
highly specific and efficient way to silence, edit, or insert genetic
components” stated Co-founder
Under the terms of the agreement, bluebird bio issued the former
stakeholders of Pregenen 408,667 shares of bluebird common stock at
closing, and paid or assumed approximately
Conference Call and Webcast
bluebird bio will host a conference call at
About Homing Endonucleases (HE) – also called Meganucleases (MN)
Homing Endonucleases (HE) are the most specific naturally occurring DNA cleaving enzymes yet discovered, employing an integrated DNA binding and cleavage mechanism to target DNA sequences greater than 20 base pairs in length. HEs are compact, non-repetitive, and use highly efficient cleavage chemistry, thus enabling “multiplex” gene editing: multiple genomic sequences can be targeted for modification using a single gene delivery vector. This offers the potential for improved efficacy by unlocking multiple mechanisms of action within gene and cell based therapies. HE’s unique biology promotes the editing or replacement of defective genes by leveraging the body’s natural cellular DNA repair mechanisms.
MegaTALs are a single-chain fusion enzyme that combines the natural DNA cleaving processes of Homing Endonucleases (HEs) with the DNA binding region of transcription activator-like (TAL) effectors. TALs are easily engineered proteins that recognize specific DNA sequences. This protein fusion architecture allows the generation of extremely active and highly specific and compact nucleases that are compatible with all current viral and non-viral cell delivery methods.
About bluebird bio, Inc.
bluebird bio is a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases. bluebird bio has two clinical-stage programs in development. The most advanced product candidate, Lenti-D, is in a recently-initiated phase 2/3 study, the Starbeam Study, for the treatment of childhood cerebral adrenoleukodystrophy (CCALD), a rare, hereditary neurological disorder affecting young boys. The next most advanced product candidate, LentiGlobin, is currently in two phase 1/2 studies, one in the US (the Northstar Study) and one in
bluebird bio also has an early-stage chimeric antigen receptor-modified
T cell (CAR-T) program for oncology in collaboration with
bluebird bio has operations in
This release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the potential for Pregenen’s gene editing technology platform, bluebird bio’s plans with respect to advancing product candidates using that platform, the payment of future milestone payments to former Pregenen stakeholders, as well as bluebird bio’s existing product candidates and research programs. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that the Pregenen technology platform will not successfully identify new or viable product candidates, that the preliminary results from our clinical trials will not continue or be repeated in our ongoing clinical trials, the risk that previously conducted studies involving similar product candidates will not be repeated or observed in ongoing or future studies involving current product candidates, the risk of cessation or delay of any of the ongoing or planned clinical studies and/or our development of our product candidates, the risk of a delay in the enrollment of patients in our clinical studies, the risk that our collaboration with
Availability of other information about bluebird bio
Investors and others should note that we communicate with our investors and the public using our company website (www.bluebirdbio.com), our investor relations website (http://www.bluebirdbio.com/investor-splash.html), including but not limited to investor presentations and FAQs,
Source: bluebird bio, Inc.
bluebird bio, Inc
Richard E. T. Smith, Ph.D., 339-499-9382
Pure Communications, Inc.
Dan Budwick, 973-271-6085