> ---------- Forwarded message ---------- > From: Marie <magpie...@gmail.com> > Date: Thu, 21 Dec 2017 09:46:47 -0800 > Subject: [blindphones] Attention to > people with RP > To: blindpho...@groups.io > > I am including some information I > received yesterday concerning a gene > therapy treatment which has been > approved by the FDA for people with > Retinitis Pigmentosa. I am sending it > to all the lists I currently > belong to because it needs to get to as > many RP sufferers as possible. > Marie > Foundation Fighting Blindness > Celebrates Historic FDA Approval of > > First Gene Therapy to Treat Blindness > > Foundation’s early investment in > LUXTURNA™ boosts vision-restoring > treatment for people with RPE65 > mutations and will help advance other > gene therapies currently in > development. > > (Columbia, MD) — Today’s U.S. Food > and Drug Administration (FDA) > approval of voretigene neparvovec, to > be marketed as LUXTURNA, will be > life-changing for patients with vision > loss due to mutations in the > RPE65 gene and a watershed moment for > the inherited retinal disease > field, says the Foundation Fighting > Blindness. The Foundation was an > important early investor in LUXTURNA, > providing $10 million in > critical seed funding for the therapy. > > The groundbreaking treatment is the > first gene therapy for the eye and > for any inherited disease to be > approved by the FDA. The treatment > restores vision by delivering working > copies of the RPE65 gene > directly into the retina, thereby > compensating for the nonfunctional, > mutated genes. > > “We are thrilled for the patients > whose lives will change dramatically > because of this treatment,” says > David Brint, Foundation Fighting > Blindness chairman. “We are also > pleased to have this concrete example > of the strength of the Foundation’s > strategy of identifying and > investing early in promising > treatments. Doing so helps attract > industry investment that can usher > promising treatments through > clinical trials and ultimately FDA > approval.” > > LUXTURNA is the result of more than two > decades of research and > development at the University of > Florida, the University of > Pennsylvania, Children’s Hospital of > Philadelphia, and Spark > Therapeutics. The Foundation Fighting > Blindness’ seed investment > allowed researchers to take the therapy > through the early > investigational stages critical to any > treatment development. > > “LUXTURNA will be life-changing for > people with an inherited retinal > disease caused by RPE65 mutations. For > them, the treatment means alife > of independence. Also important is the > momentum this approval provides > to other gene-based therapies — for > the eye and other diseases — now > in the clinic,” says Benjamin Yerxa, > PhD, Foundation CEO. > > An additional noteworthy milestone is > the demonstrated value of a new > clinical endpoint devised by the Spark > Therapeutics team to measure > LUXTURNA’s impact. The new measure, a > multi-luminance mobility test > (informally called the maze), measured > the impact of the treatment > beyond the traditional visual acuity > measure — the eye chart. This new > clinical endpoint moves vision measures > beyond the eye chart, which is > particularly significant for people > with low or no vision. > > Spark Therapeutics, which holds the > biologics license for LUXTURNA and > conducted the clinical trials that > showed its safety and efficacy, > will also manage the treatment rollout. > Spark has announced that in > order to ensure the treatment is safely > administered, it will only be > available through a small number of > centers of clinical excellence > across the country. Spark has also > expressed its commitment to > educating third-party payers about the > value of LUXTURNA and to > working to help ensure treatment access > to all eligible patients. > > Anyone in need of more information > about LUXTURNA should contact Spark > Therapeutics at 1-833-SPARK-PS > (833-772-7577). Another resource for > information is Spark’s website: > www.Sparktx.com. > > > Foundation Fighting Blindness > Celebrates Historic FDA Approval of > > First Gene Therapy to Treat Blindness > > Foundation’s early investment in > LUXTURNA™ boosts vision-restoring > treatment for people with RPE65 > mutations and will help advance other > gene therapies currently in > development. > > (Columbia, MD) — Today’s U.S. Food > and Drug Administration (FDA) > approval of voretigene neparvovec, to > be marketed as LUXTURNA, will be > life-changing for patients with vision > loss due to mutations in the > RPE65 gene and a watershed moment for > the inherited retinal disease > field, says the Foundation Fighting > Blindness. The Foundation was an > important early investor in LUXTURNA, > providing $10 million in > critical seed funding for the therapy. > > The groundbreaking treatment is the > first gene therapy for the eye and > for any inherited disease to be > approved by the FDA. The treatment > restores vision by delivering working > copies of the RPE65 gene > directly into the retina, thereby > compensating for the nonfunctional, > mutated genes. > > “We are thrilled for the patients > whose lives will change dramatically > because of this treatment,” says > David Brint, Foundation Fighting > Blindness chairman. “We are also > pleased to have this concrete example > of the strength of the Foundation’s > strategy of identifying and > investing early in promising > treatments. Doing so helps attract > industry investment that can usher > promising treatments through > clinical trials and ultimately FDA > approval.” > > LUXTURNA is the result of more than two > decades of research and > development at the University of > Florida, the University of > Pennsylvania, Children’s Hospital of > Philadelphia, and Spark > Therapeutics. The Foundation Fighting > Blindness’ seed investment > allowed researchers to take the therapy > through the early > investigational stages critical to any > treatment development. > > “LUXTURNA will be life-changing for > people with an inherited retinal > disease caused by RPE65 mutations. For > them, the treatment means alife > of independence. Also important is the > momentum this approval provides > to other gene-based therapies — for > the eye and other diseases — now > in the clinic,” says Benjamin Yerxa, > PhD, Foundation CEO. > > An additional noteworthy milestone is > the demonstrated value of a new > clinical endpoint devised by the Spark > Therapeutics team to measure > LUXTURNA’s impact. The new measure, a > multi-luminance mobility test > (informally called the maze), measured > the impact of the treatment > beyond the traditional visual acuity > measure — the eye chart. This new > clinical endpoint moves vision measures > beyond the eye chart, which is > particularly significant for people > with low or no vision. > > Spark Therapeutics, which holds the > biologics license for LUXTURNA and > conducted the clinical trials that > showed its safety and efficacy, > will also manage the treatment rollout. > Spark has announced that in > order to ensure the treatment is safely > administered, it will only be > available through a small number of > centers of clinical excellence > across the country. Spark has also > expressed its commitment to > educating third-party payers about the > value of LUXTURNA and to > working to help ensure treatment access > to all eligible patients. > > Anyone in need of more information > about LUXTURNA should contact Spark > Therapeutics at 1-833-SPARK-PS > (833-772-7577). Another resource for > information is Spark’s website: > www.Sparktx.com. Disclaimer: 1. 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