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

Genes & Cancer | Now on PubMed: Volume 12

Scienmag

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

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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.

FDA sets out advice to developers of gene editing medicines

Bio Pharma Dive

Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing

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

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

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

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

Pfizer expands into gene editing with Beam research deal

Bio Pharma Dive

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

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

A biotech startup launches with $38M to develop a cystic fibrosis gene therapy

Bio Pharma Dive

Carbon Biosciences believes its technology, which is based on parvoviruses, could allow for larger gene delivery and repeat dosing

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

Intellia, expanding in gene editing, buys startup Rewrite for $45M

Bio Pharma Dive

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

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 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.

ACMG releases update to secondary findings gene list; SF v3.1 adds five genes, including one with variant linked to heart failure in underrepresented populations

Scienmag

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).

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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

Vertex aligns with another CRISPR gene editing startup

Bio Pharma Dive

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

New method boosts the study of regulation of gene activity

Scienmag

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

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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.

Moderna, teaming with a startup, wades further into gene editing

Bio Pharma Dive

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

Beam, Apellis partner in deal to expand gene editing's reach

Bio Pharma Dive

The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing

Novel gene for Alzheimer’s disease in women identified

Scienmag

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.

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Gene linked to severe learning disabilities governs cell stress response

Scienmag

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. –

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Gene therapy for hemophilia: So close, yet so far away

Bio Pharma Dive

Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety

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

Vertex, CRISPR strengthen case for pioneering gene-editing treatment

Bio Pharma Dive

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

Scientists unravel the mystery of genes that are key to brain development

Scienmag

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

Record funding flowed into cell, gene therapy companies last year

Bio Pharma Dive

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

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 editing biotech Verve to raise $267M in large IPO

Bio Pharma Dive

Capitalizing on investor interest in gene editing, the high-profile startup has secured one of the richest biotech initial public offerings priced this year

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

Changed gene expression after heart surgery extends cardiomyocyte regeneration

Scienmag

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.

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