Brainstorm Health: Spark Gene Therapy, Meatless Meat, Flu Vaccine and Egg Allergies

Share
Brainstorm Health: Spark Gene Therapy, Meatless Meat, Flu Vaccine and Egg Allergies

Luxturna is a single injection that works by delivering 150 billion viral vector particles containing a correct copy of the RP65 gene to retinal cells, restoring the patient's ability to make the missing enzyme.

Federal health officials have approved the nation's first gene therapy for an inherited disease, a treatment that improves the sight of patients with a rare form of blindness.

The FDA Wednesday approved Spark Therapeutics Inc.'s voretigene neparvovec-rzyl (Luxturna), the first gene therapy for inherited vision loss caused by faulty gene mutations.

It's the first gene therapy approved in the United States for a disease caused by mutations in a specific gene, and only the third gene therapy ever approved. The condition affects between 1,000 and 2,000 people in the United States.

Spark said Tuesday that it won't be releasing the price until early next year. As reported by the Associated Press, Lovelace, from Kentucky, was among several patients urging the FDA to approve the treatment. "Spanning the course of ten years, the research conducted at CHOP's Center for Cellular and Molecular Therapies laid the groundwork for this revolutionary gene therapy". High was joined by Jean Bennett, MD, PhD, F.M. Kirby professor of Ophthalmology at the Perelman School of Medicine at the University of Pennsylvania's Scheie Eye Institute, and Albert M. Maguire, MD, a professor of Ophthalmology at Penn Medicine and an attending physician at CHOP, who served as a Principal Investigator of the therapy's clinical trials.

Next year the agency plans to issue guidance documents that will provide instructions for companies developing gene therapies for specific diseases, including "more efficient parameters" than now exist. "There are more than 250 genes involved in vision and we hope that this may pave the way for the development of treatment for other inherited retinal diseases/causes of congenital blindness".

In the Phase 3 study, 27 of 29 treated patients had meaningful improvement in their ability to see, according to the Lancet report. The most common adverse reactions from treatment with Luxturna included eye redness (conjunctival hyperemia), cataract, increased intraocular pressure, and retinal tear. So we can guess at why Spark didn't release theirs: the cost of this treatment is likely to be mind-boggling. Those results confirmed more than a decade's worth of research. "I believe gene therapy will become a mainstay in treating, and maybe curing, many of our most devastating and intractable illnesses", he noted.

"We're at a turning point when it comes to this novel form of therapy and at the FDA, we're focused on establishing the right policy framework to capitalize on this scientific opening", Gottlieb noted. FDA Commissioner Scott Gottlieb said in a statement.

Share

Advertisement

Related Posts

Power outage brings Atlanta airport to a standstill
Southwest Airlines told passengers to keep checking the airline's app and website to get updates on flights early Monday morning. Global flights to Hartsfield were diverted to other Atlanta-area airports, US Customs and Border Protection said.

John Legend to star in 'Jesus Christ Superstar Live!' on NBC
We always knew John Legend had the voice of an angel, but this latest announcement is the confirmation we needed. Legend reacted to Webber's tweet, writing, "Just woke up to Andrew Lloyd Webber tweeting about me".

AAA Projects Record-Breaking Holiday Travel in MA and Across the US
A record-breaking number of New Englanders are expected to travel for the year-end holidays, according to statistics from AAA. A strong economy and labor market are to thank for increased income and consumer confidence, she said.

CME Group, CBOE Bitcoin Futures Have Tepid Debuts
As big-name players continue to get involved, cryptocurrency markets could continue to see healthy growth in the months to come. Tron, for instance, is up 2,000% from its asking price at the beginning of December.

Bristol-Myers Squibb Company (BMY) And Insider Trading Activity
That's a potential 1.84 increase relative to where The Procter & Gamble Company (NYSE:PG) has been trading recently. Finally, BDO Wealth Advisors LLC acquired a new stake in Bristol-Myers Squibb in the second quarter worth $123,000.

Baird Initiates Coverage on Cimarex Energy (NYSE:XEC). What May be Next?
Capital One Financial reissued an "overweight" rating on shares of Cimarex Energy in a research note on Wednesday, November 8th. The rating was upgraded by Jefferies to "Buy" on Wednesday, May 25. (NYSE:XEC) on Thursday, December 8 to "Overweight" rating.

Today's Hot Mover - Eli Lilly and Company, (NYSE: LLY)
Following the completion of the transaction, the insider now owns 123,084,104 shares in the company, valued at $10,856,017,972.80. Shares have moved $3.75 over the past month and more recently, $0.82 over the past week heading into the earnings announcement.

After US veto, United Nations assembly to vote on Jerusalem resolution
The draft United Nations resolution calls upon all countries to refrain from establishing diplomatic missions in Jerusalem. Such a vote is non-binding, but carries political weight.

Defender reveals why he rejected a move to the Premier League
Terry ended his 19-year spell with Chelsea at the end of last season and opted to join Villa during the summer transfer window. Chelsea lie in third place in the table, 14 points behind runaway leaders Manchester City and three behind Manchester United .

Baby girl born after spending almost 25 years as frozen embryo
Jeffrey Keenan, medical director of the NEDC, said the organization was privileged to help the couple become parents. A 26-year-old Tennessee woman has given birth to a healthy girl born of a frozen embryo nearly as old as she is.

© 2015 ExpressNewsline. All Rights reserved.