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Gene 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
Scienmag
JUNE 16, 2022
BUFFALO, NY- June 16, 2022 – Oncology-focused research papers published in Volume 12 of Genes & Cancer are now on PubMed. Read short previews below about some of the groundbreaking studies published in 2021 by Genes & Cancer. Biology cancer Genes PubMed Volume
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
MARCH 16, 2022
Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing
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
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
MARCH 30, 2022
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
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
JANUARY 10, 2022
Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases
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
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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
JUNE 21, 2022
Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing
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
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
FEBRUARY 3, 2022
Founded by University of California, Berkeley scientists, Rewrite is studying ways to expand the reach of gene editing beyond limitations of current technologies, including Intellia'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
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.
Scienmag
JUNE 17, 2022
The American College of Medical Genetics and Genomics (ACMG) has released an update to the recommended minimum gene list for the reporting of secondary findings (SF).
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 26, 2021
A new alliance with Mammoth Biosciences marks the latest evidence of Vertex's interest in gene editing, which has already been the focus of multiple deals with emerging biotechs
Scienmag
JULY 1, 2022
One way cells can control the activities of their genes is by adding small chemical modifications to the DNA that determine which genes are turned on or off. Researchers have found that in bacteria DNA methylation plays a role in regulating virulence, reproduction and gene […]. Latest News activity boosts gene method regulation study
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
NOVEMBER 2, 2021
The biotech has identified gene editing as a natural fit for the technology it uses to deliver its vaccines and drugs. A deal with Metagenomi, a startup backed by Bayer, will further those ambitions
Bio Pharma Dive
JUNE 30, 2021
The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing
Scienmag
JUNE 30, 2022
Latest News Alzheimers Disease gene identified women(Boston)—Alzheimer’s disease (AD) is a progressive neurodegenerative disorder and the most common cause of dementia, affecting more than 5.8 million individuals in the U.S.
Scienmag
MAY 27, 2022
A gene that has been associated with severe learning disabilities in humans has been found to also play a vital role in cells’ response to environmental stress, according to a Duke University study appearing May 24 in the journal Cell Reports. A gene that […]. DURHAM, N.C. –
Bio Pharma Dive
JANUARY 28, 2021
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety
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
JUNE 11, 2022
Positive data from 75 patients with either sickle cell disease or beta thalassemia keep the companies’ therapy on track to become the first CRISPR-based treatment submitted to drug regulators, possibly by the end of the year
Scienmag
JUNE 16, 2022
Scientists are starting to understand the precise workings of a type of gene that, unlike other genes, does not code for proteins – the building blocks of life. Medicine & Health brain development Genes key mystery scientists unravel
Bio Pharma Dive
MARCH 16, 2021
Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM
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
Bio Pharma Dive
JUNE 17, 2021
Capitalizing on investor interest in gene editing, the high-profile startup has secured one of the richest biotech initial public offerings priced this year
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
Scienmag
JUNE 24, 2022
Biology cardiomyocyte changed expression extends gene heart regeneration SurgeryBIRMINGHAM, Ala. While lower vertebrates can repair their adult hearts after a heart attack, mammals — including humans — cannot.
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
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