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Gene Therapy Related Topics 
Bio Pharma Dive
JUNE 24, 2022
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
Bio Pharma Dive
JANUARY 13, 2022
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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Bio Pharma Dive
JUNE 8, 2022
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
Bio Pharma Dive
MAY 20, 2022
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
Bio Pharma Dive
MARCH 24, 2022
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
Bio Pharma Dive
JUNE 28, 2022
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.
Bio Pharma Dive
APRIL 4, 2022
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
Bio Pharma Dive
APRIL 1, 2022
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
Bio Pharma Dive
MAY 17, 2022
An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials
Bio Pharma Dive
APRIL 28, 2022
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
Bio Pharma Dive
JANUARY 8, 2021
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth
Bio Pharma Dive
FEBRUARY 4, 2022
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
Bio Pharma Dive
SEPTEMBER 3, 2021
A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field
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Bio Pharma Dive
DECEMBER 8, 2021
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
Bio Pharma Dive
MARCH 8, 2022
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
Bio Pharma Dive
JULY 22, 2021
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
Bio Pharma Dive
OCTOBER 12, 2021
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
Bio Pharma Dive
NOVEMBER 3, 2021
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
Bio Pharma Dive
JUNE 27, 2022
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.
Bio Pharma Dive
JUNE 23, 2022
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
Bio Pharma Dive
JUNE 9, 2022
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
Camargo
JULY 27, 2021
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.
Bio Pharma Dive
MAY 17, 2022
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
Bio Pharma Dive
DECEMBER 2, 2020
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
Bio Pharma Dive
JUNE 15, 2021
Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study
Bio Pharma Dive
MAY 31, 2022
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
Bio Pharma Dive
JUNE 10, 2022
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
Bio Pharma Dive
MAY 21, 2021
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
Bio Pharma Dive
OCTOBER 22, 2020
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
Bio Pharma Dive
APRIL 1, 2021
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
Bio Pharma Dive
MAY 3, 2022
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
Bio Pharma Dive
SEPTEMBER 1, 2021
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
Bio Pharma Dive
OCTOBER 7, 2021
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
Bio Pharma Dive
MAY 25, 2022
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
Bio Pharma Dive
APRIL 26, 2022
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
Bio Pharma Dive
NOVEMBER 15, 2021
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
Bio Pharma Dive
JANUARY 4, 2022
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
Bio Pharma Dive
NOVEMBER 5, 2020
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
Bio Pharma Dive
SEPTEMBER 7, 2021
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
pharmaphorum
JUNE 27, 2022
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.
Bio Pharma Dive
JULY 23, 2021
Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy
Bio Pharma Dive
JULY 1, 2021
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
Bio Pharma Dive
JULY 28, 2021
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
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