Sparks therapeutics? I don't see any big-name research hospitals like Johns Hopkins or other retina foundation based clinics. Little strange. No technical details here.
Deidre > On Dec 22, 2017, at 1:13 AM, Mohib Anwar Rafay <[email protected]> wrote: > > just wondering what will be the expensis incurred for this treatment? > >> On 12/21/17, Marie <[email protected]> wrote: >> 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. >> >> >> --- >> This email has been checked for viruses by Avast antivirus software. >> https://www.avast.com/antivirus >> >> -- >> The following information is important for all members of the V iPhone >> list. >> >> If you have any questions or concerns about the running of this list, or if >> you feel that a member's post is inappropriate, please contact the owners or >> moderators directly rather than posting on the list itself. >> >> Your V iPhone list moderator is Mark Taylor. Mark can be reached at: >> [email protected]. 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