Breaking Gene Therapy News

Gene-Therapy Firm UniQure Explores Options Including Sale - Yahoo Finance

Jun 16, 2019 | 15:51 pm

Gene-Therapy Firm UniQure Explores Options Including Sale Yahoo Finance(Bloomberg) -- UniQure NV is exploring options including a potential sale amid interest from pharmaceutical companies looking to expand in gene therapy, ...

Is $1.8 Million for bluebird bio's New Gene Therapy Ridiculous? - Fox Business

Jun 15, 2019 | 23:00 pm

Is $1.8 Million for bluebird bio's New Gene Therapy Ridiculous? Fox BusinessFollowing European approval earlier this month, bluebird bio announced its beta-thalassemia gene therapy will launch with a sky-high price tag.

Gene Therapy: A comfortable case of Boogie fever - Pittsburgh Post-Gazette

Jun 15, 2019 | 13:00 pm

Gene Therapy: A comfortable case of Boogie fever Pittsburgh Post-GazetteAs I understand it, Stevie Wonder came into a place called the Square Club in Braddock one night and asked to sit in with the Precisions, a singing group put ...

Bluebird Bio Stock Falls as Sales of Gene Therapy Are Delayed. - Barron's

Jun 14, 2019 | 22:00 pm

Bluebird Bio Stock Falls as Sales of Gene Therapy Are Delayed. Barron'sNew Gene Therapy Priced at $1.8 Million in Europe The Wall Street JournalBluebird Bio Sets $1.8M Price on Gene Therapy for Rare Blood Disease XconomyBluebird Bio CEO defends $1.8 million price for gene therapy — 'It's really thinking about it differently' CNBCBluebird prices gene therapy at 1.58 million euros over 5 years ReutersView full coverage on Google News

Bluebird's gene therapy will cost $1.8 million. Cue the price debate - STAT

Jun 14, 2019 | 14:03 pm

Bluebird's gene therapy will cost $1.8 million. Cue the price debate STATZynteglo, the one-and-done gene therapy for beta thalassemia newly approved in Europe, will carry a price tag of $1.8 million, Bluebird announced.

SMA Gene Therapy Zolgensma 'Saved All of Our Lives': A Family's Story - SMA News Today

Jun 14, 2019 | 14:00 pm

SMA Gene Therapy Zolgensma 'Saved All of Our Lives': A Family's Story SMA News TodayTina and Torence Anderson share the story of their son, Malachi, who has SMA type 1 and became one of the first babies treated with Zolgensma.

CRISPR gene therapy could advance using "jumping genes" - Axios

Jun 13, 2019 | 23:38 pm

CRISPR gene therapy could advance using "jumping genes" AxiosTwo prominent teams of scientists recently announced transposons — or "jumping genes" — can improve the precision of CRISPR gene editing. Why it matters: ...

Gene therapy costs, manufacturing keeping CBER head 'up at night' - BioPharma Dive

Jun 13, 2019 | 20:00 pm

Gene therapy costs, manufacturing keeping CBER head 'up at night' BioPharma DivePeter Marks, head of the Food and Drug Administration's Center for Biologics Evaluation and Research, said his top concern for gene therapy's future is ...

MOF-encapsulated DNA for gene therapy - Nanowerk

Jun 13, 2019 | 09:04 am

MOF-encapsulated DNA for gene therapy NanowerkA critical challenge in gene therapy is the safe and effective delivery of genetic materials across cell membranes into target cells. Although viral systems have ...

Measles Madness And Value: Gene Therapy, Prevention, And The Pre-Vaccine Era - Health Affairs

Jun 12, 2019 | 14:25 pm

Measles Madness And Value: Gene Therapy, Prevention, And The Pre-Vaccine Era Health AffairsThe value of keeping healthy through prevention is often distorted relative to the value treatment offers individuals who are suffering devastating illnesses.

How will we pay for potentially curative gene therapies? - STAT

Jun 12, 2019 | 10:46 am

How will we pay for potentially curative gene therapies? STATWe have arrived at a special moment in health care. Innovative, life-changing gene therapies are here that will cure or ease debilitating diseases. Yet these ...

Could automating raw materials production bring down gene therapy prices? - MedCity News

Jun 10, 2019 | 22:04 pm

Could automating raw materials production bring down gene therapy prices? MedCity NewsExperts at last week's BIO meeting in Philadelphia discussed the long-term implications of many gene therapies hitting the market, and ways their prices could ...

Costly new gene therapy developed at Nationwide Children's gives families hope - The Columbus Dispatch

Jun 9, 2019 | 11:00 am

Costly new gene therapy developed at Nationwide Children's gives families hope The Columbus DispatchDonovan Weisgarber sits at the piano, slowly raising his tiny arms above his head before striking the keys as hard as he can. “Again!” the 3-year-old exclaims.

The gene therapy era has arrived. So have the challenges. - BioPharma Dive

Jun 7, 2019 | 09:00 am

The gene therapy era has arrived. So have the challenges. BioPharma DiveA landmark regulatory approval has vaulted the cell and gene therapy space to the front of the drug industry's mind. But speedy progress has also brought ...

Parkinson's: New gene therapy shows promise for prevention - Medical News Today

Jun 7, 2019 | 09:00 am

Parkinson's: New gene therapy shows promise for prevention Medical News TodayResearchers from Japan have tested a new gene therapy against Parkinson's disease using mouse models. Their results suggest that the approach is effective.

Gene Therapy Appears to Be Beating Once-Incurable Diseases - Bloomberg

Jun 5, 2019 | 09:00 am

Gene Therapy Appears to Be Beating Once-Incurable Diseases BloombergGene therapy appears to successfully treat once-incurable patients like “bubble boy” Omarion Jordan. But progress like this doesn't come cheap.

Five Companies That Are Changing the Landscape for Cell and Gene Therapy - BioBuzz

Jun 4, 2019 | 09:00 am

Five Companies That Are Changing the Landscape for Cell and Gene Therapy BioBuzzJune 4th, 2019. Gene therapies represent some of the most promising advancements in science and human health in recent history. The BioHealth Capital ...

Europe approves first gene therapy to treat rare blood disease - STAT

Jun 3, 2019 | 09:00 am

Europe approves first gene therapy to treat rare blood disease STATBluebird Bio's one-time gene therapy won approval in Europe, marking a major milestone and a chance to learn from the field's past commercial mistakes.

Germline gene therapy: Safety - Science Daily

Jun 3, 2019 | 09:00 am

Germline gene therapy: Safety Science DailyAn internationally known embryologist and his son make the case for using gene-editing tools to prevent inherited disease, in a new editorial. The authors push ...

Gene therapy gets FDA approval -- and a $2 million price tag - CNN

May 24, 2019 | 09:00 am

Gene therapy gets FDA approval -- and a $2 million price tag CNNThe US Food and Drug Administration approved a treatment Friday for a genetic disease called spinal muscular atrophy that causes infants' muscles to waste ...

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