Gene Therapy: Page 17
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Pfizer expands into gene editing with Beam research deal
Pfizer's work with mRNA vaccines led it to explore other applications of the technology, resulting in a multiyear partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases.
By Ned Pagliarulo • Jan. 10, 2022 -
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Sanofi cuts ties with Sangamo, sharpening focus on 'off-the-shelf' cell therapy
The French pharma cited a "change in strategic direction" for ending a long-running pact focused on genetic medicines for blood diseases, led by a sickle cell treatment in early testing.
By Ben Fidler • Jan. 6, 2022 -
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, researchers and drugmakers still face major challenges.
By BioPharma Dive staff -
Novartis ups investment in gene therapy for the eye with $800M buyout
The deal for Gyroscope Therapeutics, worth up to $1.5 billion, gives the Swiss pharma access to a treatment for a type of age-related blindness as well as new means of delivering drugs into the eye.
By Jonathan Gardner • Dec. 22, 2021 -
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Duchenne patient dies in Pfizer gene therapy study
The tragic news follows changes Pfizer recently made to the design of another study testing the therapy due to side effects seen in some participants.
By Jonathan Gardner • Dec. 21, 2021 -
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Bluebird hit with another hold for sickle cell gene therapy
Study enrollment and treatment of sickle cell patients under 18 will be paused for safety reasons, another hurdle in a series of setbacks for the company.
By Ned Pagliarulo • Dec. 20, 2021 -
Spark expands in Philadelphia, with new facility meant to be Roche's gene therapy hub
The Luxturna developer, now owned by Roche, will invest $575 million to build a huge research and development center on Drexel University's campus.
By Ned Pagliarulo • Dec. 17, 2021 -
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Pfizer opens gene therapy plant in $800M North Carolina expansion
The new facility cost nearly $70 million to build, and is part of a major push by the pharma giant to become a leader in genetic medicine manufacturing.
By Kristin Jensen • Dec. 16, 2021 -
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Rebounding from setbacks, Sanofi reveals promising data for hemophilia drug
Fitusiran, which Sanofi licensed from Alnylam, could finally get to market after trial delays and safety concerns slowed its progress. But gene therapies and other new medicines might provide competition.
By Jonathan Gardner • Dec. 14, 2021 -
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Fresh data show UniQure's hemophilia gene therapy appears to hold up
The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.
By Jacob Bell • Dec. 9, 2021 -
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A high-profile gene therapy biotech takes aim at Huntington's
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.
By Jacob Bell • Dec. 8, 2021 -
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Deep Dive // Gene editingHeart attacks struck Sek Kathiresan’s family. He’s devoted his life to stopping them.
After several family members had early heart attacks, Kathiresan vowed to understand why they happen. His research journey has changed medical practice and led to a new biotech startup, Verve Therapeutics, that seeks to prevent them.
By Ben Fidler • Nov. 29, 2021 -
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Bluebird, after delays, gets speedy FDA review for beta thalassemia gene therapy
The regulator will decide whether to approve Bluebird's treatment by next May, but its evaluation is beginning more than a year later than the biotech hoped.
By Ned Pagliarulo • Nov. 22, 2021 -
Permission granted by Ed Shipman for Mass Eye and Ear
Deep Dive // Gene therapy safetyYears later, a first-of-its-kind treatment shows the power, and limits, of gene therapy
Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it.
By Shoshana Dubnow • Nov. 15, 2021 -
Courtesy of Sanofi
Eye-focused gene therapy startup gets $60M cash infusion from Sanofi
Shortly after postponing a $135 million IPO, Gyroscope has found another way to back a treatment for a disease that's become a top target of multiple drugmakers.
By Jonathan Gardner • Nov. 8, 2021 -
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Beam gets green light to begin first clinical test of base editing
Alongside the milestone, the gene editing biotech also announced John Maraganore, who will soon step down as Alnylam's CEO, will join its board.
By Ned Pagliarulo • Nov. 8, 2021 -
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Blackstone continues biotech push with up to $250M cell therapy investment
The investment in CAR-T maker Autolus Therapeutics adds to a string of biotech deals the private equity firm has made since 2018.
By Ben Fidler • Nov. 8, 2021 -
Jeenah Moon via Getty Images
Pfizer gene therapy research delayed by trial changes, safety questions
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.
By Ned Pagliarulo • Nov. 3, 2021 -
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FDA extends review of J&J, Legend's cell therapy for multiple myeloma
The three-month delay could give an advantage to Bristol Myers Squibb, which holds the first approval for a CAR-T therapy for the blood cancer.
By Jonathan Gardner , Ben Fidler • Nov. 2, 2021 -
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Moderna, teaming with a startup, wades further into gene editing
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.
By Ben Fidler • Nov. 2, 2021 -
NIH, FDA spearhead broad partnership to speed gene therapy research
The two institutions will work with drugmakers and nonprofit groups on a wide-ranging initiative meant to make the complex treatments easier and less costly to produce.
By Kristin Jensen • Oct. 28, 2021 -
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Vertex aligns with another CRISPR gene editing startup
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.
By Ben Fidler • Oct. 26, 2021 -
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Bluebird, winding down in Europe, withdraws another rare disease gene therapy
The biotech won EU approval for two gene therapies, Zynteglo and Syksona, both of which it will pull from market after difficulties negotiating reimbursement.
By Ned Pagliarulo • Oct. 21, 2021 -
Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy
Drugs from Vertex can effectively treat the lung disease in as much as 90% of patients. Gene therapy could be a solution for the remaining 10%, but the path forward is challenging.
By Shoshana Dubnow • Oct. 19, 2021 -
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. 7, 2021
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