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

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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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

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

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

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.

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

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

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

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

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

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

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

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

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

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

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

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.

UniQure buoyed by early data for Huntington's gene therapy

Bio Pharma Dive

After one year, researchers detected important protein changes in patients who received a low dose of the experimental treatment. Further testing and functional data are needed to assess its potential, however

FDA panel backs Bluebird gene therapy despite safety risks

Bio Pharma Dive

Advisers to the agency voted 15-0 in support of the rare disease treatment eli-cel, which was linked to a bone marrow cancer in three study participants

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.

Ultragenyx strikes deal with struggling Abeona for rare disease gene therapy

Bio Pharma Dive

The treatment, which is designed to treat Sanfilippo syndrome, could become Ultragenyx's first gene therapy to reach market in the U.S., according to the biotech's CEO

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

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 delays planned FDA filing for hemophilia gene therapy

Bio Pharma Dive

BioMarin now expects to resubmit its approval application to the FDA by the end of September, the latest regulatory setback for the company's closely watched treatment

FDA advisers offer unanimous support to second Bluebird gene therapy

Bio Pharma Dive

By a 13-0 vote, the panel agreed the benefits of Bluebird’s beta thalassemia treatment outweigh its risks, one day after reaching a similar conclusion for another of the company’s drugs

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

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

Audentes CEO exits as Astellas rebrands a gene therapy buyout

Bio Pharma Dive

Natalie Holles, who was appointed CEO of Audentes soon after Astellas acquired the gene therapy developer in 2019, has departed amid the company's integration

Pfizer cleared to restart hemophilia gene therapy trial

Bio Pharma Dive

While a voluntary pause in dosing new patients will remain in place, the FDA's decision puts the study back on track to deliver data in 2023

What to expect at the FDA's two-day meeting on gene therapy safety

Bio Pharma Dive

A group of advisers convened by the regulator will spend Thursday and Friday weighing a range of safety worries with gene therapy, and how best to prevent them

Intergalactic Therapeutics launches with $75M to build a new type of gene therapy

Bio Pharma Dive

Founded by former Biogen executive Michael Ehlers and backed by Apple Tree Partners, Intergalactic aims to create non-viral gene therapies delivered via an advanced form of electroporation

FDA starts clock on review of CSL, UniQure gene therapy

Bio Pharma Dive

The regulator agreed to assess the companies' data under priority review, setting up a decision on approval of the hemophilia B treatment in six months

Undeterred by gene therapy's struggles, a startup launches into a downturn

Bio Pharma Dive

Apertura Gene Therapy, a new company formed by Deerfield, is hoping a suite of next-generation tools will help it stand out at a time when this area of research has fallen out of favor with some public investors

Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy

Bio Pharma Dive

Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it

Avrobio stops work on rare disease gene therapy after unexpected study results

Bio Pharma Dive

The biotech's treatment was one of the furthest along in testing for Fabry, a rare inherited disease that's become a target for gene therapy developers

Gene therapy for Duchenne muscular dystrophy: Nearing final tests

Bio Pharma Dive

Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Bio Pharma Dive

On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice

BioMarin gene therapy for haemophilia on course for EU approval

pharmaphorum

The EMA’s human medicines advisory committee has recommended that BioMarin Pharmaceutical’s haemophilia A gene therapy Roctavian be approved in the EU, removing one of the last barriers to launch.

A biotech's eye gene therapy faces longer odds as serious side effects pile up

Bio Pharma Dive

Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy

The 'dovetail' partnership meant to turn Boston Children's into a hub for gene therapy startups

Bio Pharma Dive

A five-year alliance with high-profile biotech ElevateBio is designed to get around a bottleneck that has held back the institution’s gene therapy work

A biotech startup raises $117M to deliver gene therapies in a new way

Bio Pharma Dive

Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector