Ashley and Cole Carper traveled from Little Rock, AR, to tell their family’s story at the FDA hearing.
With five gene-therapy clinical trials underway or soon to begin, Applied Genetic Technologies Corporation (AGTC) is generating tremendous excitement for the potential to overcome vision loss from several inherited retinal diseases.
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.
The National Center for Excellence in Fighting Blindness, a Gund-Harrington Initiative, has opened its fourth annual Gund-Harrington Scholar Award competition to support innovative research efforts that seek to prevent, treat or cure blindness.
Labels in English are (clockwise): Intelligent neuromorphic camera/LED; Matrix for optical stimulation of neurons/electronic; Implant for data processing/external; Antenna for energy transfer and wireless communication; External module for visual information transfer
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.
Translational research — moving promising science out of laboratories and into clinical trials — is essential to getting vision-saving, retinal-disease treatments out to the millions who need them. With that said, translational research is also costly and high risk and requires extensive clinical development and regulatory knowledge.