Opus Genetics Receives Rare Pediatric Disease Designation from the U.S. FDA for Ocular Gene Therapy OPGx-LCA5 to Treat Rare Inherited Retinal Disease LCA5
August 20 2024 - 8:00AM
Opus Genetics, a patient-first, clinical-stage gene therapy company
developing treatments for inherited retinal diseases, today
announced the U.S. Food and Drug Administration (FDA) has granted
Rare Pediatric Disease designation (RPD) for its ocular gene
therapy OPGx-LCA5 to treat patients with the inherited retinal
disease LCA5. OPGx-LCA5 is an adeno-associated virus 8 (AAV8)
vector designed to precisely deliver a
functional LCA5 gene to the outer retina in patients with
Leber congenital amaurosis (LCA) resulting from biallelic mutations
in the LCA5 gene (LCA5).
“We are thrilled to receive Rare Pediatric Disease designation
from the FDA for our OPGx-LCA5 gene therapy. This important
milestone brings us closer to delivering a potential treatment for
patients with LCA5,” said Ben Yerxa, Ph.D., chief executive officer
of Opus. “At Opus, we’re committed to advancing therapies that help
treat patients with inherited retinal diseases, and this
designation further validates the potential impact of our
innovative ocular gene therapy approach. We look forward to
providing updates on the Phase 1/2 clinical trial evaluating
OPGx-LCA5 soon.”
LCA5 is a form of early-onset retinal degeneration that
affects approximately one in 1.7 million people in the U.S.
Currently, there are no approved treatments for individuals with
LCA5-related vision loss. OPGx-LCA5 is currently being studied in
an open-label, dose-escalation Phase 1/2 clinical trial at the
University of Pennsylvania designed to evaluate its safety and
preliminary efficacy in 15 patients with inherited retinal
degeneration due to biallelic mutations in the LCA5 gene. For more
information on the trial, visit clinicaltrials.gov
(NCT05616793).
The FDA grants Rare Pediatric Disease designation to
therapeutics intended to treat serious or life-threatening rare
diseases that primarily affect individuals under the age of 18. By
obtaining this designation, Opus has access to valuable incentives
and support from the FDA during its development program. With this
designation, OPGx-LCA5 will be eligible to receive a priority
review voucher upon approval for any subsequent marketing
application that can be sold or transferred to other companies.
About OPGx-LCA5OPGx-LCA5 is designed to address
a form of Leber congenital amaurosis (LCA) due to biallelic
mutations in the LCA5 gene (LCA5), which encodes the
lebercilin protein. LCA5 is an early-onset severe
inherited retinal dystrophy. Studies in LCA5 patients
have reported evidence for the dissociation of retinal architecture
and visual function in this disease, suggesting an opportunity for
therapeutic intervention through gene augmentation. OPGx-LCA5 uses
an adeno-associated virus 8 (AAV8) vector to precisely deliver a
functional LCA5 gene to the outer retina. OPGx-LCA5 is
currently being evaluated in a Phase 1/2 clinical trial at the
University of Pennsylvania designed to evaluate its safety and
preliminary efficacy in 15 patients with inherited retinal
degeneration due to biallelic mutations in the LCA5 gene.
About Opus GeneticsOpus Genetics is a
clinical-stage gene therapy company for inherited retinal diseases
with a unique model and purpose. Backed by Foundation Fighting
Blindness’ venture arm, the RD Fund, Opus combines unparalleled
insight and commitment to patient need with wholly owned programs
in numerous orphan retinal diseases. Its AAV-based gene therapy
portfolio, including a derisked LCA5 lead program currently in a
Phase 1/2 clinical trial, tackles some of the most neglected forms
of inherited blindness while creating novel orphan manufacturing
scale and efficiencies. Based in Research Triangle Park, N.C., the
company leverages knowledge of the best science and the expertise
of pioneers in ocular gene therapy to transparently drive
transformative treatments to patients. For more information, visit
www.opusgenetics.com.
Media Contact:Gina Mangiaracina6 Degrees
gmangiaracina@6degreesPR.com