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Posts tagged gene therapies

MeiraGTx Treats First Patient in XLRP Gene-Therapy Trial

MeiraGTx, a gene-therapy company in London and New York City, has treated its first patient in a gene-therapy clinical trial for people with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. The Phase I/II study is taking place at Moorfields Eye Hospital in London. The safety-oriented trial will enroll 36 participants. Three dose levels of the therapy will be evaluated.

XLRP is a leading cause of inherited, progressive retinal degeneration and vision loss. The condition usually affects males, but is also diagnosed occasionally in females. Mutations in the gene RPGR cause about 70 percent of XLRP cases. RPGR mutations affect about 15,000 people in the United States and tens of thousands more around the world.
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Foundation Fighting Blindness and 4D Molecular Therapeutics Partner to Boost Retinal Gene Therapy Development

Promising gene therapies for inherited retinal diseases (IRDs) — many of which have moved into clinical trials — use viral delivery systems (aka vectors) to get copies of the therapeutic gene to the retinal cells that need them. Viruses are well suited when used for gene delivery, because they’re adept at penetrating cells with their genetic cargo. While some viruses in their natural state can cause illness, therapy developers remove the native genes and replace them with those that are therapeutic.

The Foundation Fighting Blindness and 4D Molecular Therapeutics (4DMT), an innovative leader in viral vector development, have formed a partnership to help companies and researchers quickly obtain and implement high-quality vectors for their retinal gene-therapy development efforts.
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