Gene Therapy

FT: GlaxoSmithKline Considers Splitting Group

GlaxoSmithKline Plc is considering a break-up of the group after investors pressed the company's board to spin off its consumer division, the …

GlaxoSmithKline

PTC Therapeutics to Acquire Agilis Biotherapeutics

<b>- Expands and diversifies current pipeline with four gene therapy programs -</b><p><b>- BLA submission in AADC deficiency expected in 2019 -</b><p>SOUTH PLAINFIELD, …

Administrative Law

Gene Editing Is Coming: Get In Front Of Sangamo Therapeutics's 'Irrational Extrapolation Event'

Moby Dick<p>There is a fascinating chase for finding companies that the market, due to its necessity to discount all potential outcomes, cannot properly …

Business

Scientists treated a mouse’s genetic disorder in the womb — here’s what’s next

The downside of working with fetuses is that the mom is also involved<p>This week, scientists reported that they were able to treat a serious genetic disorder in the womb — in mice. It sounds like science fiction, but this type of treatment may not be far away for humans. What will we need to figure …

Genetics

PTC Therapeutics stock spikes on $200 million acquisition of Agilis Biotherapeutics

PTC Therapeutics PTCT, +4.46% said Thursday afternoon that it plans to acquire gene-therapy startup Agilis Biotherapeutics for $200 million plus …

S&P 500 Index

PTC Therapeutics to acquire Agilis Biotherapeutics for $200M; shares up 8.5%

PTC Therapeutics (NASDAQ:PTCT) agrees to acquire gene therapy developer Agilis Biotherapeutics for $200M in cash and stock.<p>Agilis' lead candidate is …

Administrative Law

Biotech With 'Superstar' Drug Also Plans To Buy Gene Therapy Firm

<b>PTC Therapeutics</b> (PTCT) stock spiked late Thursday after the biotech company said it plans to acquire gene therapy player Agilis Biotherapeutics.<p>X<p>In …

Business

Liz Parrish Wants to Live Forever

She flew down early on a Monday without telling her husband or kids where she was going or what she was doing. By the time she landed in Bogotá it was late. The sky was hazy and dark, the air surprisingly cool, the roads crowded and chaotic.<p>Her hosts had prepared a bed at their apartment, but she …

Telomere

Newsletter

NATURE PODCAST<p>Listen to the latest science news, brought to you by Benjamin Thompson and Noah Baker.<p>Subscribe<p>iTunesAcastRSS<p>This week, tougher DNA …

Chemistry

Genprex To Present At The Singular Research Summer Solstice 2018 Conference

NEW YORK, July 18, 2018 /PRNewswire/ -- Genprex, Inc. (GNPX), a clinical stage gene therapy company developing a new approach to treating cancer …

Cancer Cells

Gold rush to gene-editing in the brain

Research Highlight | Published: 18 July 2018<p>GENETIC ENGINEERING<p>Michelle Trenkmann<p><i>Nature Reviews Genetics</i> (2018) | Download …

Google Scholar

Biotech IPO Boom Comes With Side Effects

Biotech stocks are hot tickets once again on Wall Street and that is bringing a wave of high-risk, high-reward initial public offerings.<p>Small and midsize biotech stocks are having a terrific year. The S&P Biotechnology Select Industry Index has hit record highs, up about 15% so far this …

Business

DNA Damage from CRISPR Has Been ‘Seriously Underestimated’

The breakthrough gene therapy has hit some major stumbling blocks in the last few months that threaten its status as a therapy.<p><i>This article is part of</i> DNA/IDK <i>, a semi-regular column exploring how genetic modification is shaping the future.</i> <b>Follow along here</b> <i>.</i><p>When CRISPR was first revealed to the …

Genetics

Voyager advancing gene therapy for Parkinson's, Phase 2 data may be sufficient for U.S. marketing application

Based on FDA feedback, Voyager Therapeutics (NASDAQ:VYGR) says it maintains its plan to submit a BLA seeking approval for gene therapy VY-AADC based …

Business

Pfizer's Hemophilia B Gene Therapy Candidate Enters Phase III

1 / 2<p>Pfizer (PFE) initiates phase III program on a gene therapy treatment, fidanacogene elaparvovec, for patients with hemophilia B.<p><b>Pfizer Inc. PFE</b> …

Genetics

Pfizer launches late-stage study of hemophilia B gene therapy

<i>0</i><p>Recent Alerts<p>Sign up to create alerts for Instruments, Economic Events and content by followed authors<p>Free Sign Up<p>Already have an account? Sign …

Genetics

Fetal gene therapy prevents fatal neurodegenerative disease

A fatal neurodegenerative condition known as Gaucher disease can be prevented in mice following fetal gene therapy, finds a new study led by UCL, the …

Medicine

Bellicum finance chief to bid adieu

Bellicum Pharmaceuticals (NASDAQ:BLCM) CFO Alan Musso will depart effective August 31. VP of Finance and Controller Rosemary Williams will serve as …

Business

Axovant Sciences Failed To Mention Critical Information

On July 10th 2018 Axovant Sciences (AXON) announced a partnership with Benitec Biopharma (BNTC) for exclusive global rights to BB-301, now named …

Business

Janney head cheerleader for BioLife

Janney analyst Paul Knight is bullish on BioLife Solutions (BLFS +0.5%), maintaining his Buy rating on the stock citing its strong Q2 performance and …

Business

Type 2 diabetes, obesity may soon be reversed with gene therapy

New research shows that gene therapy can completely reverse markers of type 2 diabetes and obesity in rodents.<p>The prevalence of diabetes, or the …

Diabetes

Faster, Electric Gene Therapy Could Cure Diseases

A stunning new study published in the journal <i>Nature</i> on Wednesday shows that researchers have figured out a potentially faster, more efficient way to …

Molecular Biology

3 Gene Therapy Companies In Hot Pursuit Of A 'One-And-Done' Cure

3:35 PM ET<p>Related news<p>3:15 PM ET In the stock market, growth investors stand a greater chance of significant profits when they focus on companies …

Business

FDA lays down the law on gene therapy, offering guidances on both diseases and procedures

As first announced in January, the FDA’s Center for Biologics Evaluation and Research (CBER) on Wednesday offered a swath of new draft guidance …

Clinical Trials

MedImmune, 4D Molecular Therapeutics to collaborate on gene therapy

AstraZeneca subsidiary and California-based firm to develop AAV-based therapy for chronic liver disease.

Health Care

REGENXBIO adds to up move, shares ahead 3%

REGENXBIO (RGNX +2.9%) is up on below-average volume. Shares have more than tripled since early April when Novartis announced its $8.7B takeout of …

Business

Axovant, Benetic Jump on Agreement to Develop Gene Therapy for MD

Axovant Sciences Ltd. (AXON) knows how to kick off the week right.<p>Shares of the biopharmaceutical company rose 5% in early morning trading Monday …

ALS

Gene Therapy: BioMarin, Spark Can Both Win Treating Hemophilia

By<p>July 9, 2018 11:30 a.m. ET<p>Gene therapy has been hailed as a potential breakthrough treatment for many diseases, and today <b>Credit Suisse's Martin</b> …

Molecular Biology

Orchard Therapeutics Appoints Joanne Beck, Ph.D. to Board of Directors

BOSTON and LONDON, July 09, 2018 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a leading commercial-stage biotech company dedicated to transforming the …

Los Angeles

Gene therapy shown to cure type 2 diabetes and obesity in mice, researchers report

A single administration of a therapeutic vector in mouse models cures type 2 diabetes and obesity in the absence of long-term side effects, …

Diabetes