According to a story from World Pharma Today, Andelyn Biosciences, Inc., which is a gene therapy Contract Development and Manufacturing Organization (CDMO), has recently struck a partnership with Odylia Therapeutics,…
Medscape reported that when Victoria Gray was three months old, her family was told to keep her close: her prognosis wasn't looking good. She had been rushed to the ER…
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…
NPR News recently featured an article announcing that scientists at Portland’s Casey Eye Institute have attempted to use CRISPR to edit a gene with the DNA still in place.…
In a story for Drug Development & Delivery, the CEO of the biotechnology company ProQR, Daniel de Boer, recently sat down for an interview. In this interview, Mr. de Boer…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is a concept a few decades…
Genes themselves are a relatively new discovery, found only about a century ago. Gene therapy, a medical procedure that alters genes to address disease, is in its first 30 years.…
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Clustered Regularly Interspaced Short Palindromic Repeats is more commonly known as CRISPR. As reported recently in Science News, the CRISPR/Cas9 “molecular scissors” is slated to make its highly anticipated debut…
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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…
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 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 story from EurekAlert!, a collaborative team of researchers from the University of Florida and The Salk Institute are using cryo-electron microscopy in order to more closely examine…
The FDA, US Food and Drug Administration, unanimously agreed upon the recommendation for a new gene therapy that treats a rare form of blindness, reported ScienceNordic. Spark Therapeutics, a biotech…
Gene therapy has been on the horizon for years now, but before today, it was only a reality for patients with non-inherited diseases caused by genetic mutations. That just changed!…
On this season of America's Got Talent, the world met Christian Guardino. I was one of millions who heard his beautiful singing voice as he belted out his song onstage.…
The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA). MeiraGTx received rare pediatric disease designation for its…
By examining a zebrafish, scientists have found the gene that affects the maintenance of cone photoreceptors and visual functions in the eye. It might be the knowledge that’s been needed…
Case Western Reserve University researchers have recently developed gene-carrying nanoparticles that are able to bring healthy genes to those negatively affected by Leber congenital amaurosis (LCA), a disease that causes…
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