Gene Therapy: Page 18
-
Getty Images
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 -
Getty Images
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 -
Explore the Trendline➔
Getty Images
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 -
Getty / Edited by BioPharma Dive
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 -
Seth Babin/BioPharma Dive
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 -
Getty Images
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 -
Getty Images
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 -
Kena Betancur via Getty Images
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 -
Mario Tama via Getty Images
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 -
Maddie Meyer via Getty Images
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 -
Mario Tama via Getty Images
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 -
Getty / Edited by BioPharma Dive
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 -
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. 6, 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. 4, 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 -
Getty / Edited by BioPharma Dive
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 -
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 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 -
Getty / Edited by BioPharma Dive
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
Previous
