In a press release from early March 2021, cell therapy company CiRC Biosciences, Inc. ("CiRC") shared that its chemically induced photoreceptor-like cells (CiPCs) received Orphan Drug designation from the FDA.…
Recently, biopharmaceutical company GenSight Biologics ("GenSight") announced the publication of data from a study evaluating GS030-Drug Products (GS030-DP or "GS030") as a potential treatment option for retinitis pigmentosa (RP). Although…
Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…
Researchers from Nanoscope, thanks to funding by the NIH, have developed a protein that can effectively restore vision in mice. This study was published in Nature Gene Therapy. The Study…
For patients who lose their vision, artificial and implantable devices like retinal prostheses offer a way forward. But while these prostheses are already somewhat effective, a Korean research team recently…
Janssen Pharmaceuticals has an ongoing Phase 1/2 clinical trial for the inherited retinal disease X-linked retinitis pigmentosa (XLRP). They are investigating an adeno-associated gene therapy called RPGR as a therapeutic…
As reported in Biospace, a new gene therapy has made notable progress towards curing x-linked retinitis pigmentosa, a disease responsible for sight loss in men. Until now, the disease irreversibly…
Personalized Medicine You may have heard of personalized medicine. It's a new wave of healthcare that aims to provide individualized treatment to patients based on their own genetic makeup. The…
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Allergan plc, a leading global pharmaceutical company, and its partner, Editas Medicine, Inc. have been given the green light for a clinical trial designed to treat patients with severe…
According to a story from globenewswire.com, the drug developer ProQR Therapeutics N.V. has recently announced that the US Food and Drug Administration (FDA) has officially cleared the company's Investigational New…
May is Healthy Vision Month! Rare disease or not, maintaining healthy eye health should be a priority for all of us. The National Institute of Health has resources for not…
ACHM and XLRP Achromatopsia (ACHM) is a rare, inherited retinal disease which causes impaired cone photoreceptor function. Patients experience light sensitivity so extreme that it can cause blindness during the…
Ocugen Pharmaceuticals has just received their first ever orphan drug designation by the FDA for a gene therapy treatment. It's called OCU400 and it's being investigated as a treatment for…
According to a story from pm360online.com, the drug developer Novartis has recently announced that the company's gene therapy Luxturna has gained approval in the EU as a treatment for Leber's…
According to a story from BioPortfolio, the biopharmaceutical company GenSight Biologics recently announced that the first patient has received treatment in a Phase I/II clinical trial testing the company's experimental…
Nightstar Therapeutics has released preliminary data from a Phase 1/2 study of an experimental gene therapy designed to treat X-linked retinitis pigmentosa. For more detailed information you can view the…
According to a story from Eye on the Cure, the biopharmaceutical company Ophthotech is hard at work researching and developing gene therapy solutions for a variety of rare retinal conditions.…
According to a report by Newswise, a new form of gene therapy has been developed which may slow or prevent retinitis pigmentosa. Researchers funded by the National Eye Institute (NEI)…
Bruce Howell is 63 years old living with retinitis pigmentosa. He’s been legally blind since age 37. As a result, public transportation is a critical service to him. Without the…
Researchers at Columbia University have found a way to apply CRISPR, a tool used in gene therapies, to new types of disease, reports News Medical. The study was carried out…
When Kristen Smedley learned her son, Michael, was diagnosed with retinitis pigmentosa, she decided she wouldn't sit back and watch it take away from her son's life, she would take…
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