CAMBRIDGE, Mass. & SEATTLE--(BUSINESS WIRE)--bluebird bio, Inc. (Nasdaq: BLUE), a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and orphan diseases, today announced the acquisition of Precision Genome Engineering, Inc., or Pregenen, a privately held biotechnology company headquartered in Seattle, Washington. Pregenen is a leader in the development of gene editing and cell signaling technologies with a broad range of potential therapeutic applications.
“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 Nick Leschly, chief bluebird. “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.”
“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 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.”
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 $4.9 million of current liabilities of Pregenen and its stakeholders. The former stakeholders of Pregenen are also eligible to receive up to an additional $15.0 million in cash upon the achievement of certain preclinical milestones as well as $20.1 million in cash upon achievement of certain clinical milestones and $99.9 million in cash upon the achievement of certain commercial milestones with respect to product candidates identified using Pregenen’s technology.
Conference Call and Webcast
bluebird bio will host a conference call at 4:30 pm EDT on Monday, June 30, 2014 to discuss the transaction. Investors may listen to the webcast of the conference call live on the "Calendar of Events" section of bluebird bio’s website, www.bluebirdbio.com. Alternatively, investors may listen to the call by dialing: (844) 825-4408 from locations in the U.S. and (315) 625-3227 from outside the U.S. The webcast replay will be available for at least 72 hours following the call.
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.
About MegaTALs
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 France (HGB-205), for the treatment of
beta-thalassemia major. The phase 1/2 HGB-205 study also allows
enrollment of patient(s) with sickle cell disease, and bluebird bio is
planning a separate U.S. sickle cell disease trial (HGB-206).
bluebird bio also has an early-stage chimeric antigen receptor-modified T cell (CAR-T) program for oncology in collaboration with Celgene Corporation.
bluebird bio has operations in Cambridge, Massachusetts and Paris, France. For more information, please visit www.bluebirdbio.com.
About Precision Genome Engineering, Inc. (Pregenen)
Precision
Genome Engineering, Inc., or Pregenen, is a Seattle-based,
privately-held biotechnology company designing and engineering molecules
critical to a new generation of gene and cell-based therapies for unmet
medical needs. Pregenen has developed a proprietary DisplayArray™
platform to reprogram TrueCut™ Homing Endonucleases and related enzymes
highly specific to single genomic targets. These enzymes are combined
with other key technologies to modify cells and optimize their
therapeutic potential. Pregenen has also developed a novel cell
signaling system designed to provide pharmacological control over
chimeric antigen receptor driven immunotherapies. Pregenen’s lead
programs are in pre-clinical development. Pregenen was founded by
researchers at the Seattle
Children’s Research Institute, the Fred
Hutchinson Cancer Research Center and the University
of Washington.
Forward-Looking Statements
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 Celgene will not
continue or will not be successful, and the risk that any one or more of
our product candidates will not be successfully developed and
commercialized. For a discussion of other risks and
uncertainties, and other important factors, any of which could cause our
actual results to differ from those contained in the forward-looking
statements, see the section entitled “Risk Factors” in our most recent
quarterly report on Form 10-Q, as well as discussions of potential
risks, uncertainties, and other important factors in our subsequent
filings with the Securities and Exchange Commission. All information in
this press release is as of the date of the release, and bluebird bio
undertakes no duty to update this information unless required by law.
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, Securities
and Exchange Commission filings, press releases, public conference calls
and webcasts. You can also connect with us on Twitter @bluebirdbio, LinkedIn
or our YouTube
channel. The information that we post on these channels and websites
could be deemed to be material information. As a result, we encourage
investors, the media, and others interested in bluebird bio to review
the information that we post on these channels, including our investor
relations website, on a regular basis. This list of channels may be
updated from time to time on our investor relations website and may
include other social media channels than the ones described above. The
contents of our website or these channels, or any other website that may
be accessed from our website or these channels, shall not be deemed
incorporated by reference in any filing under the Securities Act of 1933.