Astellas takes stake in gene therapy developer Taysha

Bio Pharma Dive

Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders

5 questions facing gene therapy in 2022

Bio Pharma Dive

As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines

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Replay reveals second gene therapy spinout, debuting new biotech Telaria

Bio Pharma Dive

Telaria is the second spinout in Replay’s “hub-and-spoke” model, and will focus on developing a gene therapy for recessive dystrophic epidermolysis bullosa

Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting

Bio Pharma Dive

Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test

BioMarin's hemophilia gene therapy recommended for approval in Europe

Bio Pharma Dive

The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival

Next-level quality control in cell and gene therapy

Bio Pharma Dive

Discover examples of how Droplet Digital™ PCR (ddPCR™) is enabling cell and gene therapies to reach further than ever before

FDA approves first gene therapy for hemophilia B

Bio Pharma Dive

The treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million

‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy

Bio Pharma Dive

Gene therapies could help treat many ultra-rare diseases. But they may not get developed if drugmakers can’t build a sustainable business around them, CBER director Peter Marks said at a conference

As biotech retreats, gene therapy companies retrench and redraw plans

Bio Pharma Dive

At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn

5 questions facing gene therapy in 2021

Bio Pharma Dive

Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth

Viral vector design for gene therapies

Bio Pharma Dive

Safety issues in clinical trials cast doubt on the ability of gene therapy to safely mitigate disease, prompting further research into understanding the virus-host interaction that resulted in new generations of viral vectors

Mainstreaming cell and gene therapy – Realizing its potential

Bio Pharma Dive

The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles

Orchard turns to layoffs in cutting gene therapy research

Bio Pharma Dive

The biotech company plans to reduce its workforce by 30%, or by about 65 employees, in the latest example of a gene therapy developer restructuring

Astellas, despite recent setbacks, opens $100M gene therapy plant

Bio Pharma Dive

The opening of the plant, which could create more than 200 new jobs, comes on the heels of several significant issues with the company’s most advanced gene therapy program

At FDA meeting, gene therapy experts wrestle with field's blindspots

Bio Pharma Dive

A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field

Yet another gene therapy developer turns to layoffs

Bio Pharma Dive

Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines

PTC's first gene therapy gains European regulator's backing

Bio Pharma Dive

The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year

A high-profile gene therapy biotech takes aim at Huntington's

Bio Pharma Dive

Passage Bio and the University of Pennsylvania's Gene Therapy Program said they have begun preclinical research aimed at simultaneously developing multiple approaches to treating Huntington's

In England, Orchard secures rare agreement on gene therapy coverage

Bio Pharma Dive

Gene therapy developers have struggled to secure reimbursement in Europe, making Orchard's deal with the National Health Service in England and Wales a notable accomplishment

Gene therapy for Fabry: early stages, promising results

Bio Pharma Dive

Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon

Lilly bets more than $600M on a gene therapy developer targeting hearing loss

Bio Pharma Dive

Like with Prevail Therapeutics, Lilly’s first gene therapy acquisition, the pharma’s planned buyout of Akouos includes a contingent value right that could hike the deal’s overall cost

Scientists home in on cause of Duchenne gene therapy side effect

Bio Pharma Dive

An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials

After setbacks, a gene therapy company mulls a sale

Bio Pharma Dive

Sio Gene Therapies, formerly known as Axovant, said it's focused on conserving cash, and has decided to significantly reduce its workforce and terminate licensing agreements on its last two pipeline programs

Novartis turns to Voyager for nervous system gene therapies

Bio Pharma Dive

The deal, which carries a $54 million upfront payment, could give Novartis access to as many as five of Voyager's capsids, which would be used to develop gene therapies for the central nervous system

Takeda takes aim at a biotech's gene therapy work

Bio Pharma Dive

For an upfront payment of $45 million, Takeda gains access to as many as eight programs from Poseida Therapeutics and a potential method of delivering gene therapies without the help of viruses

Navigate the complexities of cell and gene therapies

Bio Pharma Dive

See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product

Pfizer gene therapy research delayed by trial changes, safety questions

Bio Pharma Dive

Data readouts for Pfizer's hemophilia gene therapies are now expected later than previously forecast, while the company also works through modifying a trial of its Duchenne treatment

Gene Therapy and Pharmacokinetics

Camargo

How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19.

J&J pushes further into eye gene therapy

Bio Pharma Dive

The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy

Novartis reports deaths of two patients treated with Zolgensma gene therapy

Bio Pharma Dive

The deaths were due to acute liver injury, a known risk of Zolgensma and a concern for gene therapies like it

Gene therapy approvals bring validation as field closes year on high

Bio Pharma Dive

It’s been a busy fall in gene therapy, with FDA nods for new treatments from CSL and Bluebird as well as a few deals. Catch up on our best stories here

BioMarin signals lengthier FDA review for hemophilia gene therapy

Bio Pharma Dive

The agency wants to see forthcoming study results for the biotech's treatment, which could potentially push out an approval decision by three months

Astellas makes another gene therapy play, takes stake in Taysha

Pharma Phorum

Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates.

Biogen gene therapy deal has yet to bear fruit

Bio Pharma Dive

Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study

BioMarin resubmits its hemophilia gene therapy to the FDA

Bio Pharma Dive

The resubmission has been long awaited after BioMarin’s original application was unexpectedly rejected by the FDA two years ago. The company expects a decision around the middle of next year

J&J-backed gene therapy for the eye clears early study test

Bio Pharma Dive

biotech MeiraGTx, could rebuild expectations after another gene therapy from Biogen failed in the retinal disease Positive results for the treatment, which was developed by U.K.

Sarepta, continuing its gene therapy push, helps launch a startup

Bio Pharma Dive

The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million

Bluebird's next gene therapy gets backing from European regulator

Bio Pharma Dive

The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world

Novartis sales of Zolgensma gene therapy slow as market shifts

Bio Pharma Dive

Treatment is now mostly of infants newly diagnosed with spinal muscular atrophy, as many of those previously eligible either already received Zolgensma or other drugs from Biogen and Roche

Another Astellas gene therapy trial paused by FDA after side effect report

Bio Pharma Dive

Both of the pharma’s clinical-stage gene therapies are now on hold The regulator suspended testing of Astellas’ Pompe disease treatment after one patient experienced peripheral nerve damage.

Sangamo presses ahead with Fabry disease gene therapy

Bio Pharma Dive

Preliminary results from a Phase 1 study show Sangamo’s treatment to be safe and suggest it is working as intended, leading the biotech to move into the trial’s next phase

Bluebird gene therapy approved by FDA for rare blood disease

Bio Pharma Dive

The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a very high cost of $2.8 million in the U.S.

FDA grants speedy review to Sarepta’s Duchenne gene therapy

Bio Pharma Dive

The agency will decide on an accelerated approval of Sarepta’s treatment by May 29, months before results are expected from a potentially confirmatory Phase 3 trial