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Gene Related Topics 
Bio Pharma Dive
OCTOBER 24, 2022
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
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
NOVEMBER 21, 2022
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
Bio Pharma Dive
NOVEMBER 4, 2022
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
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
OCTOBER 3, 2022
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
Bio Pharma Dive
JULY 25, 2022
Discover examples of how Droplet Digital™ PCR (ddPCR™) is enabling cell and gene therapies to reach further than ever before
Bio Pharma Dive
JULY 20, 2022
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
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
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
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
NOVEMBER 22, 2022
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
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
NOVEMBER 30, 2022
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
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
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
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
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
JULY 20, 2022
The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease
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
NOVEMBER 28, 2022
The challenges biopharma companies face in running clinical trials and tips on how to surmount those obstacles
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
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
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
OCTOBER 18, 2022
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
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
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
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
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
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
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
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
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
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 30, 2021
The companies aim to develop therapies for complement-driven diseases, a specialty of Apellis and relatively new research ground for gene editing
Bio Pharma Dive
AUGUST 11, 2022
The deaths were due to acute liver injury, a known risk of Zolgensma and a concern for gene therapies like it
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
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
DECEMBER 6, 2022
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
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
NOVEMBER 28, 2022
See how the effective use of CMC expertise helped guide a biopharma company’s development of a CGT product
Bio Pharma Dive
OCTOBER 31, 2022
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
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