Gene Therapy
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Takeda takes aim at a biotech's gene therapy work
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.
By Jacob Bell • Oct. 12, 2021 -
Permission granted by Intergalactic Therapeutics
Intergalactic Therapeutics launches with $75M to build a new type of gene therapy
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.
By Ned Pagliarulo • Oct. 07, 2021 -
Explore the Trendline➔
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TrendlineGene Therapy
Rapid scientific advances have put the gene therapy field at the forefront of biomedical research. But, as recent setbacks have shown, reserachers and drugmakers still face major challenges.
By BioPharma Dive staff -
Jeenah Moon via Getty Images
Gene therapy developer gets chance at a comeback with Pfizer deal
Voyager Therapeutics recently scrapped much of its pipeline and replaced top executives. Now, a Pfizer deal on new technology gives investors hope.
By Jonathan Gardner • Oct. 06, 2021 -
Courtesy of Sarepta
Sarepta embarks on late-stage clinical trial of Duchenne gene therapy
Initiation of the Phase 3 trial is an important milestone for the biotech after earlier setbacks, as well as for patients with the inherited muscle disease.
By Shoshana Dubnow • Oct. 04, 2021 -
Getty / Edited by BioPharma Dive
Amicus, looking for a boost, sells its gene therapy work to a SPAC
The value of the biotech's growing gene therapy portfolio has been "unrecognized" by investors, said CEO John Crowley, leading to a sudden shift for an initiative that began just three years ago.
By Ben Fidler • Sept. 29, 2021 -
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Worrisome side effects lead Pfizer to narrow Duchenne gene therapy trial
Outside trial monitors have attributed three cases of severe muscle weakness to Pfizer's treatment, causing the company to change the design of its closely watched Phase 3 study.
By Ben Fidler • Sept. 28, 2021 -
Sarah Silbiger via Getty Images
FDA lifts hold on GeneTx, Ultragenyx study of Angelman therapy
The hold, which was put in place during the fourth quarter last year, came after five patients in an early trial experienced muscle weakness following treatment.
By Shoshana Dubnow • Sept. 27, 2021 -
Novartis
Novartis pushes further into gene therapy for the eye with deal for Swiss startup
The large drugmaker has acquired Arctos Medical and its preclinical research into optogenetics, an area Novartis already showed interest in with its buyout of Vedere Bio last year.
By Ned Pagliarulo • UPDATED: Sept. 23, 2021 at 12:12 p.m. -
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Fourth trial volunteer dies in Astellas gene therapy study
The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used.
By Ned Pagliarulo • Sept. 14, 2021 -
AbbVie
AbbVie bets big on a gene therapy for eye diseases
The company has agreed to pay $370 million, and possibly north of $1 billion more, to gain access to a Regenxbio treatment targeting wet AMD and other eye conditions.
By Jacob Bell • Sept. 13, 2021 -
metamorworks via Getty Images
Sponsored by YourwayCell and gene therapies' evolving temperature-controlled requirements call for specialized logistics solutions
The CGT industry is expected to grow 30% between 2019 and 2025, and the transition from clinical trials to commercial-scale production is now seen as inevitable.
By Leandro Moreira, Yourway • Sept. 13, 2021 -
Lilly joins RNA editing race with ProQR deal
An agreement with the Dutch biotech, which could be worth as much as $1.3 billion, expands Lilly's work in genetic medicine research.
By Kristin Jensen • Sept. 09, 2021 -
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Gene therapy safety
'The sky is not falling.' Cautious optimism follows FDA meeting on gene therapy risks
Wall Street analysts expect little impact on biotech developers after an inconclusive discussion last week. Yet the FDA still appears wary, as shown by a new hold on a BioMarin rare disease treatment.
By Ned Pagliarulo • Sept. 07, 2021 -
Jacob Bell / BioPharma Dive
Gene therapy safetyAt FDA meeting, gene therapy experts wrestle with field's blindspots
A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations.
By Ned Pagliarulo , Ben Fidler • Sept. 03, 2021 -
Sarah Silbiger via Getty Images
What to expect at the FDA's two-day meeting on gene therapy safety
A group of advisers convened by the agency began the two-day meeting Thursday with a discussion of cancer risk to AAV gene therapy.
By Ned Pagliarulo , Ben Fidler • Sept. 01, 2021 -
Getty / Edited by BioPharma Dive
Gene therapy safety
Astellas again hits pause on gene therapy trial
The company is holding off dosing more patients with AT132, a potential treatment for a deadly neuromuscular disease, after one recently experienced a serious adverse event in the form of unusual liver function.
By Jacob Bell • Sept. 01, 2021 -
Novartis
Novartis falls behind rivals in race to bring CAR-T to early lymphoma
The company's cancer cell therapy Kymriah failed to top standard care in patients with an aggressive form of lymphoma, a setting in which treatments from Bristol Myers Squibb and Gilead recently succeeded.
By Ben Fidler • Aug. 24, 2021 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
With new Fate data, same promise, questions surround 'natural killer' cell therapy
Though early results from two of the biotech's experimental lymphoma treatments have shown promise, it’s unclear whether they can match the potency and durability of T cell therapies.
By Ben Fidler • Aug. 20, 2021 -
Mario Tama via Getty Images
Cell, gene therapy company funding reaches new heights, despite setbacks
Developers of the complex treatments raised $14 billion between January and June, nearly three-quarters of last year's record-setting total, according to numbers compiled by the Alliance for Regenerative Medicine.
By Ned Pagliarulo • Aug. 19, 2021 -
Sarah Silbiger via Getty Images
FDA clears Rocket to resume testing gene therapy for a rare heart disease
The decision from the regulator ends a three-month study pause that had stalled Rocket's development of the Danon disease treatment.
By Ben Fidler • Aug. 16, 2021 -
Bluebird to wind down business in Europe amid gene therapy struggles
Shares in the biotech sunk in value as the company also announced new safety concerns for one of its leading programs.
By Jacob Bell • Aug. 09, 2021 -
Jacob Bell / BioPharma Dive
Gilead's Kite bets on early-stage biotech to advance allogeneic cell therapies
Appia Bio, which launched with over $50 million in Series A funding earlier this year, will provide preclinical research for two CAR-iNKT candidates, which Kite will pick up should they advance past early clinical testing.
By Shoshana Dubnow • Aug. 05, 2021 -
Courtesy of Sarepta
Sarepta adds another gene therapy to its ever-expanding pipeline
The biotech company followed through on an option deal it struck two years ago with Nationwide Children's, licensing an experimental treatment for the most common form of limb-girdle muscular dystrophy.
By Ned Pagliarulo • Aug. 04, 2021 -
Novartis
Gene therapy safetyFDA allows Novartis gene therapy trials to resume after nearly 2 year pause
After reviewing animal study data submitted by the Swiss pharma, the FDA cleared spinal injections of Zolgensma for study in older patients with SMA.
By Jonathan Gardner • Aug. 03, 2021 -
Bluebird preps for split by selling manufacturing plant to well-funded startup
The startup manufacturing specialist National Resilience, which raised $800 million when it launched last year, will pay Bluebird $110 million for the North Carolina facility, bringing its North American network up to 10.
By Kristin Jensen • July 29, 2021
