RE: Attention to people with RP

[Sieghard Weitzel]

It would be out of reach of most people in countries without health insurance 
and where there are no other funding options.
What do you think heart surgery costs or even the surgeries and care somebody 
requires after a serious accident?
Recently there was a case here in Canada (Vancouver to be exact) where a 
23-year old young lady developed "AHUS" (atypical hemolytic uremic syndrome). 
This disease affects only 1 in a Million people and while based on this 
statistic and Canada's population less than 40 people in Canada should have it, 
the number in fact is apparently about 150. In any case, there is a medication 
which helps people with this disease, but here in Canada this medication costs 
$750,000 a year. Yes, I did not mistype the zeros, just over $2,000 a day.
The story went on to say that while some health plans in some of the canadian 
provinces cover this outrageous amount, the British Columbia medical plan is 
not one of them, but inside less than 2 weeks after the story published, a 
follow-up story was published which said that in BC such expensive medication 
can be provided on a case by case bases and the young student was approved to 
get it.
If a young person with RP can be actually cured with this gene treatmenrt and 
be prevented from becoming blind the economic benefits and potential long-term 
costs probably far outweigh the 1 Million Dollar price tag.

Regards,
sieghard

-Original Message-
From: viphone@googlegroups.com [mailto:viphone@googlegroups.com] On Behalf Of 
Mohib Anwar Rafay
Sent: Friday, December 22, 2017 8:43 PM
To: viphone@googlegroups.com
Subject: Re: Attention to people with RP

1 million? It would be out of reach of a person!

On 12/22/17, Dulce Muccio Weisenborn <d...@lifedesigns-inc.com> wrote:
> NPR said perhaps one million for both eyes.
>
> -Original Message-
> From: viphone@googlegroups.com [mailto:viphone@googlegroups.com] On 
> Behalf Of Mohib Anwar Rafay
> Sent: Friday, December 22, 2017 1:13 AM
> To: viphone@googlegroups.com
> Subject: Re: Attention to people with RP
>
> just wondering what will be the expensis incurred for this treatment?
>
> On 12/21/17, Marie <magpie...@gmail.com> 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 momen

Re: Attention to people with RP

[Mohib Anwar Rafay]

1 million? It would be out of reach of a person!

On 12/22/17, Dulce Muccio Weisenborn <d...@lifedesigns-inc.com> wrote:
> NPR said perhaps one million for both eyes.
>
> -Original Message-
> From: viphone@googlegroups.com [mailto:viphone@googlegroups.com] On Behalf
> Of Mohib Anwar Rafay
> Sent: Friday, December 22, 2017 1:13 AM
> To: viphone@googlegroups.com
> Subject: Re: Attention to people with RP
>
> just wondering what will be the expensis incurred for this treatment?
>
> On 12/21/17, Marie <magpie...@gmail.com> 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.

RE: Attention to people with RP

[Dulce Muccio Weisenborn]

NPR said perhaps one million for both eyes.

-Original Message-
From: viphone@googlegroups.com [mailto:viphone@googlegroups.com] On Behalf Of 
Mohib Anwar Rafay
Sent: Friday, December 22, 2017 1:13 AM
To: viphone@googlegroups.com
Subject: Re: Attention to people with RP

just wondering what will be the expensis incurred for this treatment?

On 12/21/17, Marie <magpie...@gmail.com> 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 d

RE: Attention to people with RP

[Dulce Muccio Weisenborn]

FDA just approved the gene-editing procedure.  The company will name its price 
in January, which is thought to be a million dollars for both eyes.
-Original Message-
From: viphone@googlegroups.com [mailto:viphone@googlegroups.com] On Behalf Of 
Deidre Muccio
Sent: Friday, December 22, 2017 7:02 AM
To: viphone@googlegroups.com
Subject: Re: Attention to people with RP

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 <mohibra...@gmail.com> wrote:
> 
> just wondering what will be the expensis incurred for this treatment?
> 
>> On 12/21/17, Marie <magpie...@gmail.com> 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 i

Re: Attention to people with RP

[Deidre Muccio]

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  wrote:
> 
> just wondering what will be the expensis incurred for this treatment?
> 
>> On 12/21/17, Marie  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 

Re: Attention to people with RP

[Mohib Anwar Rafay]

just wondering what will be the expensis incurred for this treatment?

On 12/21/17, Marie  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