New Collaboration with Seattle Children’s Research Institute Targets New Immunotherapy Approaches to Acute Myeloid Leukemia
Phase 1/2 Study for Merkel Cell Carcinoma Planned with
Additional Preclinical Programs Across Both the Severe Genetic Disease and Oncology Therapeutic Areas Support IND Goals
Company Will Webcast Live from the Event Beginning at
“bluebird is at a significant inflection point, with the potential approval and launch of our first gene therapy product this year and submissions for regulatory approval for potentially three additional products through 2022,” said Nick Leschly, chief bluebird. “We have the opportunity to leverage our gene and cell therapy expertise across our platform and enable a steep innovation curve for next-generation products. We are fueled by what is just the beginning of our efforts to recode the science, systems and status quo to reach new innovation frontiers and make a significant impact on patients’ lives.”
Further strengthening its leadership position in developing transformative first-in-class and best-in-class gene and cell therapies, bluebird bio will discuss several key milestones and collaboration updates across its research pipeline, which is focused on next-generation, disruptive solutions for devastating diseases. In addition to the two clinic-ready programs planned for 2019, the company is on track to submit 1-2 investigational new drug applications in 2020 and beyond.
“Relentless innovation is in our DNA at bluebird. Our 1-to-Many research
strategy rapidly integrates and iterates our tools and technologies
across our core platforms of gene editing, gene addition and cellular
immunotherapy, to develop the next generation of gene and cell therapies
with the potential to improve patients’ lives,” said
Research highlights include:
- AML Research Collaboration with Seattle Children’s
Research Institute: The research collaboration is intended to address two challenges of tackling AML, specifically the heterogeneity of the disease as well as the salvage of normal tissues with the potential for on-target/off-tumor targeting. Our T cell immunotherapy approach is expected to leverage technology that enables T cells to target multiple antigens on the surface of cancer cells as well as bluebird’s proprietary Dimerizing Agent Regulated Immunoreceptor Complex(DARIC) platform. By utilizing the DARIC platform in potential product candidates, we expect to be able to exert pharmacologic control of CAR T cell activity in vivo, allowing the investigator to switch on and switch off the activity of the engineered T cells in the patient as needed by administering a small molecule drug. Combined with Seattle Children’s world-class bench-to-bedside expertise in the arena of oncology cell therapies, the research collaboration’s goal is to rapidly accelerate development of potential new therapies for patients with AML.
- Phase 1/2 Trial for Merkel Cell Carcinoma: An academic,
proof-of-concept phase 1/2 single-arm study evaluating Merkel Cell
Polyomavirus (MCPyV) TCR-engineered autologous T cells in combination
with avelumab (anti-PDL1) is
FDA-approved and in the final initiation stages of trial approval at the Fred Hutchinson Cancer Research Centerfor the treatment of MCC, a rare neuroendocrine cancer. Exploratory clinical data generated by researchers at the Fred Hutchinson Cancer Research Centerexploring patient derived MCPyV reactive T cells in combination with PD1 axis blockade has shown promising depth and durability of response. Results from the academic phase 1/2 single-arm study are expected to inform next-generation T cell approaches including TCR engineering and checkpoint inhibition.
- MAGE-A4: Through its collaboration with
Medigene, bluebird has developed a next-generation MAGE-A4 TCR expected to enter the clinic for solid tumors in 2020. This co-receptor-independent TCR candidate has shown robust anti-tumor activity controlling tumors in a subcutaneous melanoma xenograft model as a single agent. Moreover, this highly active TCR can be combined with bluebird’s chimeric TGF-β receptor signal converter technology to “flip” the immunosuppressive signals present in the tumor microenvironment toward T cell stimulation and proliferation. This is the first collaboration target with Medigeneof a potential six TCR products that the companies have agreed to work on together.
- Diffuse Large B-cell Lymphoma Candidate: Our DLBCL preclinical program builds on the knowledge gained from the current generation of CD19-targeting cell therapies by incorporating multiple next-generation technologies to potentially address both the depth and durability of response. Specifically, our potential DLBCL product candidate combines (i) dual-targeting directed to two novel antigens; (ii) within a unique CAR construction that is designed to enhance T cell activation; and (iii) gene editing for potential potency and durability enhancements, all in a single product candidate.
- Mucopolysaccharidosis (MPSI): Our MPSI program is focused on
the severe form of MPSI, an ultra-rare metabolic condition that causes
severe neurologic impairment and organ damage, also referred to as
Hurler Syndrome. In our academic collaboration with the
University of Minnesota, we expect to leverage key learnings from our hematopoietic stem cell lentiviral vector (HSC LVV) platform technology to deliver gene-modified cells that can potentially cross the blood-brain barrier and express high and sustained levels of therapeutic enzyme. Preclinical data in the MPS1 mouse model demonstrates full molecular correction of the disease across all critical organs impacted by the disease, including the brain, following administration of an HSC LVV-gene-modified stem cell product. These robust preclinical data support the potential clinical application of this product candidate.
To access the live webcast of bluebird bio’s Analyst Day presentation, please visit the “Events & Presentations” page within the Investors & Media section of the bluebird bio website at http://investor.bluebirdbio.com. A replay of the webcast will be available on the bluebird bio website for 90 days following the meeting.
About bluebird bio, Inc.
bluebird bio is pioneering gene therapy with purpose. From our
bluebird bio is a human company powered by human stories. We’re putting our care and expertise to work across a spectrum of disorders by researching cerebral adrenoleukodystrophy, sickle cell disease, transfusion-dependent β-thalassemia and multiple myeloma using three gene therapy technologies: gene addition, cell therapy and (megaTAL-enabled) gene editing.
bluebird bio has additional nests in
This release contains “forward-looking statements” within the meaning
of the Private Securities Litigation Reform Act of 1995, including
statements regarding the plans for the potential commercial launch of
the Company’s product candidates, as well as the advancement of the
Company’s research and development plans. 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
positive efficacy and safety results from our prior and ongoing clinical
trials of our product candidates will not continue or be repeated in our
ongoing or planned clinical trials; risks that the current or planned
clinical trials of our product candidates will be insufficient to
support future regulatory submissions or to support marketing approval
in the U.S. and EU; the risk that our collaborations with third parties
will not be successful; the risk that our planned clinical studies may
be delayed or not be initiated at all; and the risk that our product
candidates will not be successfully developed, approved or
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 Form 10-Q as well as
discussions of potential risks, uncertainties and other important
factors in our subsequent filings with the
Source: bluebird bio, Inc.