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Gene therapy - Wikipedia
In medicine, gene therapy (also called human gene transfer) is the therapeutic delivery of nucleic acid into a patient's cells as a drug to treat disease. The first ...

Gene Therapy in a Patient with Sickle Cell Disease | NEJM
Sickle cell disease results from a homozygous missense mutation in the β-globin gene that causes polymerization of hemoglobin S. Gene therapy for patients with this ...

Hematopoietic Stem-Cell Gene Therapy for Cerebral ...
BackgroundIn X-linked adrenoleukodystrophy, mutations in ABCD1 lead to loss of function of the ALD protein. Cerebral adrenoleukodystrophy is characterized by ...

Cellular & Gene Therapy Guidances
This page contains a listing of cellular and gene therapy guidances.

Could gene therapy become biotech’s growth driver in 2017 ...
Despite bouncing off a 2-year low, biotech is still an unpopular sector and investors are rightfully concerned about its near-term prospects. Recent drug failures ...

Biotech portfolio update – Jumping into gene therapy ...
After a long summer break it is time to review recent events and update the portfolio. As far as clinical readouts go, my portfolio had a brutal summer with one ...

F.D.A. Approves First Gene-Altering Leukemia Treatment ...
Emily Whitehead, shown here in May, was near death at age 6 from leukemia and became the first pediatric patient to receive the experimental gene therapy.

Lentiviral Vectors Page 2 - Biology · Kenyon College
What is gene therapy? The aim of gene therapy is to modify the genetic material of living cells for therapeutic purposes (Amado and Chen, 1999).

New Gene-Therapy Treatments Will Carry Whopping Price Tags ...
The first gene therapy treatment in the United States was approved recently by the Food and Drug Administration, heralding a new era in medicine that is ...

ONCE Stock Price - Spark Therapeutics Inc. Stock Quote (U ...
Spark Therapeutics shares rise on FDA approval of new gene therapy for blindness. Spark Therapeutics Inc. shares rose 3% in heavy morning trade Tuesday after the ...



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       *** News Filter: "Gene-Therapy"



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National Pharmaceutical Council Welcomes Four Biopharmaceutical Companies as Members
PR Newswire (press release)
Spark's technology resulted in the first FDA-approved gene therapy for a genetic disease in the U.S. The company currently has three investigational programs in clinical trials, including product candidates that have shown promising early results in ...

and more »

MedPage Today

Rewired Olympic Brains; Gene Therapy Concerns; Memory Boost from Implant
MedPage Today
Building new cerebral maps and subduing reflexes: here's how Olympic ice skaters rewire their brains to execute complex routines. (Scientific American). Sandoz's generic form of glatiramer acetate for relapsing-remitting multiple sclerosis, sold as ...


Gene Therapy for Inherited Retinal Dystrophy Gets FDA Clearance
MedPage Today
The first gene therapy for a specific mutation leading to disease was approved by the FDA, the agency announced Tuesday. Spark Therapeutics' voretigene neparvovec-rzyl (Luxturna) is now the first gene therapy approved to treat those with confirmed ...
Spark aims for availability of new gene therapy early in 2018Healio
FDA Approves Spark Therapeutics' Voretigene Neparvovec-rzylMD Magazine
Luxturna Approved to Treat Inherited Form of Vision LossMonthly Prescribing Reference
all 127 news articles »


First Successful Gene Therapy for Hemophilia A
MedPage Today
During an advance ASH media preview, Robert Brodsky, MD, of Johns Hopkins in Baltimore, included the gene therapy trial in a small group of abstracts that he characterized as "home run-type" studies. Like van den Berg, he said the impressive 52-week ...
'Promise of a Cure': Gene Therapy for Hemophilia AMedscape

all 14 news articles »

Washington Post

Spark Puts $850k Price Tag on Gene Therapy (Endpoints News
MedPage Today
The first commercial gene therapy available in the U.S. will cost $850,000, Endpoints News reports. Spark Therapeutics has set a wholesale acquisition cost of $425,000 per eye for its gene therapy Luxturna, a one-time treatment for a genetic condition ...
Price for Spark's gene therapy for blindness set at $850000Healio
Luxturna Carries Potential to Correct Blindness, $850k Price TagMD Magazine
A US drugmaker offers to cure rare blindness for $850000CNBC
Forbes -Spark Therapeutics Inc.
all 122 news articles »

DIY Gene Therapy: Biohackers Plunge Ahead (MIT Tech Review
MedPage Today
Some biohackers have started purchasing CRISPR supplies to alter their own genes. And two companies selling them plan to ignore FDA warnings that such sales are illegal, reports MIT Technology Review. The DNA editing kits vary in sophistication, from ...

and more »

Gene Therapy Offers Hope for Inherited Retinal Disorder
MedPage Today
NEW ORLEANS -- Gene augmentation by voretigene neparvovec (Luxturna) improves functional vision in patients with biallelic mutation in the RPE65 gene, a mutation that results in inherited retinal disease, researchers reported here. Twenty seven of 29 ...

Small Gene Therapy Trial Success for Rare Degenerative Brain Disease
MedPage Today
The treatment, known as Lenti-D gene therapy, involves taking a patient's own CD34+ cells and using a lentiviral vector to deliver ABCD1 complementary DNA before infusing back into the patient. Mutations in the ABCD1 gene lead to deficiencies in the ...

and more »

Ocugen obtains worldwide rights to modifier gene therapy platform
An exclusive worldwide license to develop and commercialize ophthalmology products based on technology from the Schepens Eye Research Institute of Massachusetts Eye and Ear has been obtained by Ocugen, according to a company press release. The ...

and more »



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