MeiraGTx Receives Rare Pediatric Disease Designation from FDA for AAV8-RK-RetGC for the Treatment of Patients with Leber Congenital Amaurosis due to GUCY2D Mutations

LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (NASDAQ:MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug Administration (FDA) has granted the Company Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1). This is the fourth Rare Pediatric Disease Designation the Company has received in the last three months, including AAV8-RK-AIPL1 for the treatment of LCA4 retinal dystrophy, AAV8-RK-BBS10 for the treatment of Bardet-Biedl syndrome (BBS) due to BBS10 mutations and AAV5-RDH12 for the treatment of RDH12 associated retinal dystrophy.

"Receiving Rare Pediatric Disease Designation for an additional program in our ophthalmology pipeline represents another regulatory milestone for the Company and demonstrates the groundbreaking therapeutic potential of our technology to address these severe childhood blinding conditions," said Alexandria Forbes, Ph.D., president and chief executive officer of MeiraGTx.

"As we have done with our AIPL1 program, we intend to leverage our manufacturing infrastructure and Specials License along with our clinical expertise in IRDs to work with regulators to expedite the delivery of these potentially life changing treatments to these severely affected children."

An RPDD may be granted by the FDA to drugs and biologics intended to treat certain orphan diseases affecting fewer than 200,000 patients in the U.S., the serious or life-threatening manifestations of which primarily affect individuals aged 18 years or younger. Under the FDA's Rare Pediatric Disease Priority Review Voucher (PRV) program, a sponsor that receives approval for a biologics license application for a rare pediatric disease may be eligible to receive a voucher for a priority review of a subsequent marketing application for a different product. PRVs may be used by the sponsor or sold to another sponsor for their use and have recently sold for between $100 million to $158 million.

About AAV8-RK-RetGC

Mutations in the GUCY2D gene coding for guanylate cyclase lead to severe retinal diseases in humans, with 88% of cases causing autosomal recessive Leber congenital amaurosis type 1 (LCA1) whilst heterozygous missense mutations cause autosomal dominant cone-rod dystrophy (CRD). In LCA1, photoreceptor function loss and blindness emerge very early in life. In CRD, degeneration starts in the cones and leads to loss of the central visual field due to the high presence of cones in the macula. CRD can lead to complete blindness when degeneration of rods follows those of cones.

About AAV8-RK-AIPL1

AAV8-RK-AIPL1 is an investigational genetic medicine for the treatment of one of the most severe forms of Leber congenital amaurosis (LCA) owing to genetic deficiency of Aryl-hydrocarbon-interacting protein-like 1 (AIPL1). It is delivered via subretinal injection to children, and through a one-time administration, AAV8-RK-AIPL1 is designed to deliver functional copies of the AIPL1 gene to cone and rod photoreceptors in the central retina, to slow further degeneration and restore vision.

About AAV8-RK-BBS10

The investigational genetic medicine AAV8-RK-BBS10 is an adeno-associated virus with a serotype 8 capsid with a complementary DNA (cDNA) encoding the human BBS10 gene for treatment of Bardet-Biedl syndrome (BBS) due to BBS10 mutations. BBS is a rare genetic disease affecting approximately 1 in 250,000 people around the world. One of the primary symptoms of BBS is visual impairment secondary to retinal degeneration. More than 20 different genes are associated with the development of BBS, with BBS10 accounting for approximately 25% of cases.

About AAV5-RDH12

The investigational genetic medicine AAV5-RDH12 is an adeno-associated virus serotype 5 containing the human RDH12 gene for treatment of RDH12 associated retinal dystrophy. Defects in retinol dehydrogenase 12 (RDH12) account for 3–10% of Leber congenital amaurosis (LCA) and early-onset severe retinal dystrophy (EOSRD) and is particularly devastating due to early macular atrophy. RDH12 encodes retinol dehydrogenase 12, an enzyme expressed in photoreceptors that reduces all-trans-retinal to all-trans-retinol.

About MeiraGTx

MeiraGTx (NASDAQ:MGTX) is a vertically integrated, clinical-stage genetic medicines company with a broad pipeline of late-stage clinical programs supported by end-to-end manufacturing capabilities. MeiraGTx has internal plasmid production for GMP, two GMP viral vector production facilities as well as an in-house Quality Control hub for stability and release, all fit for IND through commercial supply. In addition, MeiraGTx has developed a proprietary manufacturing platform with leading yield and quality aspects and commercial readiness, core capabilities in viral vector design and optimization and a transformative riboswitch gene regulation platform technology that allows for the precise, dose-responsive control of gene expression by oral small molecules. MeiraGTx is focusing the riboswitch platform on the delivery of metabolic peptides, including GLP-1, GIP, Glucagon, and PYY, using oral small molecules, as well as cell therapy for oncology and autoimmune diseases. MeiraGTx has developed the technology to apply genetic medicine to more common diseases, increasing efficacy, addressing novel targets, and expanding access in some of the largest disease areas where the unmet need remains high.

For more information, please visit www.meiragtx.com

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