MeiraGTx Announces Upcoming Presentations at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting
April 21 2023 - 8:30AM
MeiraGTx Holdings plc (Nasdaq: MGTX), a vertically
integrated, clinical stage gene therapy company, today announced
two abstract presentations at the Association for Research in
Vision and Ophthalmology (ARVO) 2023 Annual Meeting.
The first abstract will present humoral immune response data
from the Company’s Phase 1/2 MGT009 trial for the investigational
gene therapy botaretigene sparoparvovec (bota-vec, formerly
AAV-RPGR) in patients with the inherited retinal disease (IRD)
X-linked retinitis pigmentosa (XLRP) associated with the retinitis
pigmentosa GTPase regulator (RPGR) gene which is being developed
along with our partners Janssen Pharmaceuticals,
Inc. (Janssen), one of the Janssen Pharmaceutical Companies of
Johnson & Johnson.
The second abstract will present data from MeiraGTx’s
proprietary promoter platform involving the use of novel,
AI-assisted engineered promoters to improve gene expression in rod
photoreceptors.
“We are pleased to present recent data from the Phase 1/2
clinical study of our investigational gene therapy bota-vec at this
year’s ARVO meeting. An understanding of the immune response of
bota-vec in XLRP patients is important as we move forward in the
late stage development of this product with the aim of providing a
safe and effective treatment for patients with this irreversible
and devastating disease,” said Alexandria Forbes, Ph.D., president,
and chief executive officer of MeiraGTx. “In addition, I am very
excited to present some of the first data from our AI driven
promoter discovery platform as exemplified by the rod-specific
promoters we are presenting. Our AI driven promoter discovery
platform provides us the ability to rapidly and accurately achieve
cell specific as well as gene-appropriate expression levels in any
cell type, accelerating the development of optimized potent and
safe gene therapy vectors targeting different cell types and
tissues, including different neuronal sub-types. The rod specific
promoters presented are being applied to our optogenetic vectors
targeting rods to allow these photoreceptors to respond more like
cones, a potential treatment for central vision loss in diseases
such as Dry AMD, as well as for rod or rod-cone disorders.”
Presentation/poster #:
5446: Humoral immune response to AAV5-RPGR (botaretigene
sparoparvovec) gene therapy in RPGR-associated X-linked retinitis
pigmentosa
- April 27, 2023, 2:15-2:30pm CDT-
Session #300, Location - 353-355
CO405: AAV-based evaluation of novel in silico promoters
to drive expression in rod photoreceptors
- April 23, 2023, 12:00-1:45 pm CDT-
Session #49, Location – Exhibit Hall
The presentations will be available on the publications page of
the Company’s website after their respective presentation times.
The abstracts can be found on the ARVO Annual Meeting website.
MeiraGTx and Janssen are jointly developing bota-vec
as part of a broader collaboration to develop and commercialize
gene therapies for the treatment of inherited retinal diseases.
About botaretigene sparoparvovec
(bota-vec)Botaretigene sparoparvovec (bota-vec) is being
investigated in collaboration with Janssen for the treatment of
patients with XLRP caused by disease-causing variants in the
eye-specific form of the RPGR gene. Through a one-time
administration, bota-vec is designed to deliver functional copies
of the RPGR gene to counteract the loss of retinal cells with the
goal of preserving and potentially restoring vision for those
living with XLRP. The Phase 3 LUMEOS clinical trial (NCT04671433)
is actively dosing patients to study bota-vec for the treatment of
patients with XLRP with disease-causing variants in the RPGR gene.
Bota-vec has been granted Fast Track and Orphan Drug designations
by the U.S. Food and Drug Administration (FDA) and PRIority
MEdicines (PRIME), Advanced Therapy Medicinal Product (ATMP) and
Orphan designations by the European Medicines Agency (EMA).
About X-Linked Retinitis Pigmentosa (XLRP)XLRP
is a rare condition estimated to impact one in 40,000 people
globally.1,2 People with XLRP have progressive vision loss,
starting in childhood with night blindness.3 Over time, they lose
their peripheral vision leading to central vision loss followed by
legal blindness by the fourth decade of life.3 Currently, there are
no approved treatments for XLRP.3
About MeiraGTxMeiraGTx (Nasdaq: MGTX) is a
vertically integrated, clinical stage gene therapy company with six
programs in clinical development and a broad pipeline of
preclinical and research programs. MeiraGTx has core capabilities
in viral vector design and optimization and gene therapy
manufacturing, and a transformative gene regulation platform
technology that allows precise, dose responsive control of gene
expression by oral small molecules with dynamic range that can
exceed 5000-fold. Led by an experienced management team, MeiraGTx
has taken a portfolio approach by licensing, acquiring, and
developing technologies that give depth across both product
candidates and indications. MeiraGTx’s initial focus is on three
distinct areas of unmet medical need: ocular, including both
inherited retinal diseases as well as large degenerative ocular
diseases, neurodegenerative diseases and severe forms of
xerostomia. Though initially focusing on the eye, central nervous
system, and salivary gland, MeiraGTx plans to expand its focus to
develop additional gene therapy treatments for patients suffering
from a range of serious diseases.
For more information, please visit www.meiragtx.com.
Contacts
Investors:MeiraGTxInvestors@meiragtx.com
Media:Jason Braco, Ph.D.LifeSci
Communicationsjbraco@lifescicomms.com
1 Boughman JA, Conneally PM, Nance WE. Population genetic
studies of retinitis pigmentosa. Am J Hum Genet.
1980;32(2):223–235.2 Fishman GA. Retinitis pigmentosa. Genetic
percentages. Arch Ophthalmol. 1978;96(5):822–826.
doi:10.1001/archopht.1978.03910050428005.3 Wang DY, Chan WM, Tam
PO, et al. Gene mutations in retinitis pigmentosa and their
clinical implications. Clin Chim Acta. 2005;351(1-2):5-16.
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