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

AstraZeneca to acquire gene editing biotech LogicBio

Bio Pharma Dive

Shares in LogicBio had lost nearly all their value since a 2018 IPO, bringing the company to the brink of Nasdaq delisting. The buyout deal represents a premium of over 600

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

Roche digs deeper into gene therapy for the eye

Bio Pharma Dive

The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments

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

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

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

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

Gene editing startup iECURE returns to investors for fresh funding

Bio Pharma Dive

The company, which draws on research by Jim Wilson’s lab at UPenn, says the $65 million it raised will support the start of human testing of its lead drug through to initial clinical data

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

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

Vertex, Verve team up to develop a gene editing drug for liver disease

Bio Pharma Dive

The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease

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

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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

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

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.

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

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

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

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

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

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

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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

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

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

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

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

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

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

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

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

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

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

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

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

Replay launches gene therapy spinout Eudora, putting ‘hub-and-spoke’ plans to work

Bio Pharma Dive

The public debut of Eudora comes three months after Replay emerged with $55 million and gene delivery technology it claims can outperform AAV viral vectors