- Research in MPS IVA led by REGENXBIO scientists moving into
clinic
- Donation of NAV® Technology licenses will support
development of gene therapies for rare diseases
ROCKVILLE, Md., May 16, 2023
/PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced that
preclinical research in Mucopolysaccharidosis type IVA (MPS IVA),
also known as Morquio syndrome, was selected for inclusion in the
Foundation for the National Institutes of Health Accelerating
Medicines Partnership® Bespoke Gene Therapy Consortium
(AMP® BGTC), clinical trial portfolio. The Consortium
brings together partners such as NIH and FDA, as well as partners
from private and non-profit sectors.
Sponsored by Nemours Children's Hospital, MPS IVA is one of
eight programs selected as part of AMP® BGTC's first
clinical portfolio to help accelerate the development of bespoke
gene therapies, with the goal of streamlining the regulatory
approval process. MPS IVA is a metabolic condition that
primarily affects the skeleton, and is estimated to impact 1 in
200,000 to 300,000 individuals.
"I am proud of our scientists who led this important clinical
research work for REGENXBIO. Our mission to deliver the curative
potential of gene therapy reflects not only patients that can be
impacted by our clinical pipeline, but also all patients who should
have the opportunity to be positively impacted by gene therapy,"
said Kenneth T. Mills, President and
CEO REGENXBIO. "As a partner of the BGTC, REGENXBIO is pleased to
see this program advancing important science into the clinic."
Criteria for selection included the adequacy of the gene for
insertion into an adeno-associated virus (AAV) vector, sufficient
proof of concept and natural history data, the existence of an
established disease model, a lack of available treatment and an
overall readiness for entering into a clinical trial. REGENXBIO
will donate licenses to NAV® AAV8 and NAV®
AAV9 from its NAV® Technology Platform to enable
AMP® BGTC's development of these programs aimed at
addressing ultra rare diseases. Use of the NAV®
Technology Platform will help address the goal of making gene
therapy more accessible by creating a platform approach with
standardized processes to deliver novel therapies for many
different genetic disorders. These programs together further
validate the versatility of NAV® vectors and will
provide additional data that collectively drive the advancement of
the AAV gene therapy field.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company
seeking to improve lives through the curative potential of gene
therapy. REGENXBIO's NAV Technology Platform, a proprietary
adeno-associated virus (AAV) gene delivery platform, consists of
exclusive rights to more than 100 novel AAV vectors, including
AAV7, AAV8 and AAV9. REGENXBIO and its third-party NAV Technology
Platform Licensees are applying the NAV Technology Platform in the
development of a broad pipeline of candidates, including late-stage
and commercial programs, in multiple therapeutic areas. REGENXBIO
is committed to a "5x'25" strategy to progress five AAV
Therapeutics from our internal pipeline and licensed programs into
pivotal-stage or commercial products by 2025.
Contacts:
Dana Cormack
Corporate Communications
dcormack@regenxbio.com
Investors:
Chris Brinzey
ICR Westwicke
339-970-2843
chris.brinzey@westwicke.com
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SOURCE REGENXBIO Inc.