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AGTC seeks US FDA nod to begin phase 1/2 trial of gene therapy product candidate to treat XLRP caused by mutations in RPGR gene
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Gainesville, Florida
January 31 , 2019
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Applied Genetic Technologies Corporation (AGTC), a biotechnology company
conducting human clinical trials of adeno-associated virus (AAV)-based
gene therapies for the treatment of rare diseases, has filed an
Investigational New Drug application (IND) with the US Food and Drug
Administration to conduct a phase 1/2 clinical trial of the company's
gene therapy product candidate for the treatment of X-linked retinitis
pigmentosa (XLRP) caused by mutations in the RPGR gene.
XLRP is
an inherited condition that causes progressive vision loss, beginning
with night blindness in young boys followed by progressive constriction
of the field of vision. Affected men progress to legal blindness by
their early forties. Preclinical data indicated that treatment with an
investigational gene therapy product slowed the loss of visual function
in canines with XLRP caused by mutations in the RPGR gene. XLRP and
X-linked retinoschisis (XLRS) are the two lead development programs
within AGTC's collaboration and license agreement with Biogen, Inc., to
develop gene-based therapies for multiple ophthalmic diseases.
"We
are deeply committed to advancing therapies to address the unmet needs
of patients with rare inherited retinal disorders, and filing our fifth
IND in this field represents a significant milestone that may ultimately
improve the lives of individuals affected by XLRP," said Sue Washer,
President and CEO of AGTC. "Gene therapy clinical trials continue to
rapidly progress, making this an exciting time for AGTC, the clinical
community and patients affected by ocular conditions with no currently
available treatments."
AGTC plans to initiate a clinical study
evaluating the safety and efficacy of its proprietary gene therapy for
XLRP caused by mutations in the RPGR gene in the United States in the
coming months.
AGTC is currently scheduling patients for
enrollment in clinical trials for XLRS as well as for achromatopsia
caused by mutations in the CNGB3 and CNGA3 genes. Patients and
caregivers interested in participating in or learning about AGTC's
current and upcoming clinical trials.
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