Gene Therapy

Orchard isn't for the picking, Biohaven's Allergan headache, and who's on Vascepa?

Amarin CEO John Thero had one question for investors eager to hear about his company's plans for its heart pill, while Orchard made a case for independence.
By Jacob Bell • Jan. 16, 2020
Q&A
Novartis' David Lennon on next steps for AveXis, selling Zolgensma's price to the public

While Zolgensma's launch has gone well, the pharma still faces challenges in expanding the gene therapy's market and addressing criticism over its price.
By Andrew Dunn • Jan. 15, 2020
Nationwide Children's, a gene therapy leader, launches manufacturing spinout

Andelyn Biosciences, named after two children treated with gene therapy at Nationwide, will operate as a for-profit subsidiary of the Ohio-based hospital.
By Ned Pagliarulo • UPDATED: Jan. 15, 2020 at 10:10 a.m.
Alexion's neuro bet, Rubius' trial troubles and biotech's gene therapy milestones

On the first day of the JPM conference, Alexion mapped out ambitious plans in neuroscience, while Rubius did its best to explain delays in testing its first drug.
By Jacob Bell , Andrew Dunn • Jan. 14, 2020
BioMarin sets gene therapy sights beyond hemophilia

At the JPM conference, the biotech announced a manufacturing lift and a new trial start that affirm its ambitions in gene therapy go beyond its closely watched hemophilia A treatment.
By Jacob Bell • Jan. 13, 2020
Ultragenyx boosted by trial data for 2nd gene therapy

One analyst called the results, which sparked a 30% jump in Ultragenyx's stock price, better than expected. The biotech is now preparing a Phase 3 trial.
By Jonathan Gardner • Jan. 10, 2020
Solid cuts one-third of staff, goes all in on delayed gene therapy

Re-starting a halted muscular dystrophy trial is Solid's priority as it puts a second drug and an assistive device on the backburner.
By Jonathan Gardner • Jan. 09, 2020
Roche hands $1B to Sarepta in major return to gene therapy deals

The Swiss pharma acquired a muscular dystrophy treatment for markets outside the U.S. in a pact that could reach a value of $10 billion.
By Jonathan Gardner • Dec. 23, 2019
Muscle data doesn't help Solid build faith in its gene therapy

A regulatory delay due to an adverse event, meanwhile, has put Solid further behind Sarepta in the pursuit of a Duchenne muscular dystrophy gene therapy.
By Jonathan Gardner • Dec. 18, 2019
Homology unveils first data for PKU gene therapy, marking early milestone

Data presented Tuesday are from just three patients, but set the pace in a field that includes BioMarin and, most recently, Sangamo Therapeutics.
By Andrew Dunn • Dec. 17, 2019
Roche's $4.8B deal for Spark cleared by FTC after long delay

An unconditional OK for the deal, which was unexpectedly the target of regulatory scrutiny, should ease sector-wide concerns about acquisitions of gene therapy biotechs.
By Ned Pagliarulo • UPDATED: Dec. 17, 2019 at 7:23 p.m.
Value-based pricing for gene therapy? Maybe not ready for hemophilia

A small patient population plus large potential savings could make blood-clotting gene therapies an easier sell to payers, biotech executives say.
By Jonathan Gardner • Dec. 11, 2019
UniQure, Pfizer updates hint at gene therapy potential in hemophilia B

The two companies are advancing toward market one-time treatments for the blood disorder, with Takeda about to begin clinical testing of its own therapy.
By Jonathan Gardner • Dec. 10, 2019
Dive Awards
Drug Launch of the Year: Zolgensma

While clouded by controversy, Novartis' launch of Zolgensma shows gene therapy can be successfully commercialized, even as questions of affordability remain paramount.
By Ned Pagliarulo • Dec. 09, 2019
Sangamo keeps pressure on rivals with updated hemophilia data

Longer follow-up on Sangamo and Pfizer's gene therapy show treated patients continue to be free of bleeds, but one patient's results could spark new questions.
By Ned Pagliarulo • Dec. 07, 2019
Cancer gene therapy backed by Blackstone gets trial win

Results released Thursday are the first to be presented from a Phase 3 study of the therapy, which was recently made the cornerstone of a new company launched by Blackstone and Ferring.
By Jonathan Gardner • Dec. 05, 2019
Thermo Fisher opens $90M viral vector manufacturing plant in Massachusetts

The CDMO expects to create more than 200 jobs at the site, which is part of the company's plans to expand its gene therapy business.
By Kristin Jensen • Dec. 04, 2019
Astellas joins gene therapy race with $3B Audentes buy

Manufacturing was a key draw for Astellas, which gains access to a plant owned by Audentes that can support clinical and commercial production.
By Ned Pagliarulo • Dec. 03, 2019
Fujifilm to invest $120M in gene therapy, build center in Texas

The Japan-based company plans to spend $55 million on the center while also outfitting its joint venture with new clean rooms and bioreactors.
By Kristin Jensen • Nov. 21, 2019
The hemophilia gene therapy race faces a critical year in 2020

While BioMarin looks poised to reach market in hemophilia A first, Sangamo and Spark are advancing rival gene therapies to tackle the blood disorder.
By Andrew Dunn • Nov. 20, 2019
Takeda sees cell, gene therapy in its future. Is it too late?

Nearly a year on from completing its $62 billion Shire deal, Takeda is pitching investors on its plans to stay one of the industry's leading rare disease drug developers.
By Ned Pagliarulo • Nov. 20, 2019
Sponsored
The evolving demands of the temperature-controlled supply chain for cell and gene therapy

Cell and gene therapies are heightening the need for advanced temperature control solutions.
By Gulam Jaffer, President, Yourway • Nov. 20, 2019
First look at CRISPR, Vertex gene-editing therapy hints at treatment potential

Results from two patients, one with sickle cell disease and the other with beta-thalassemia, offer an initial glimpse at the disease-altering potential of CRISPR gene-editing.
By Ned Pagliarulo • UPDATED: Nov. 19, 2019 at 8:38 a.m.
Sarepta brings in more gene therapies with StrideBio deal

The deal adds four more neuromuscular candidates to the Exondys maker's already-rich pipeline.
By Jonathan Gardner • Nov. 14, 2019
ICER draws new gene therapy pricing framework

One-time and short-term curative therapies require a different approach to evaluating benefit, the cost watchdog group said.
By Jonathan Gardner • Nov. 13, 2019