Gene Therapy: Page 16
-
Amicus' SPAC deal unravels, leading to layoffs and cost cuts
The biotech is calling off plans to spin out its gene therapy business via a merger with a blank check company, citing unfavorable market conditions and a "challenging environment."
By Kristin Jensen • Feb. 24, 2022 -
cgtoolbox via Getty Images
Allogene, slipping in 'off-the-shelf' CAR-T race, prepares for key trials
Allogene, once the clear leader in development of a more convenient option to personalized cancer cell therapies, now is competing with CRISPR Therapeutics and other fast-emerging rivals.
By Ben Fidler • Feb. 24, 2022 -
Explore the Trendline➔
iStock via 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 -
Permission granted by Spark Therapeutics
Spark CEO Marrazzo, who led company to historic gene therapy approval, to step down
Jeffrey Marrazzo has led Spark since its founding in 2013, through the historic FDA approval of Luxturna and the company's $4.3 billion buyout by Roche.
By Ned Pagliarulo • Feb. 23, 2022 -
DKosig via Getty Images
BioMarin says gene therapy hold will endure as FDA questions cancer risk
According to BioMarin, the regulator has requested additional data to assess the "theoretical" cancer risk to the treatment, information that will take several quarters to collect.
By Ned Pagliarulo • Feb. 18, 2022 -
National Institutes of Allergy and Infectious Diseases. (2016). "Human natural killer cell" [Micrograph]. Retrieved from Flickr.
Intellia follows CRISPR peers with deal to explore 'natural killer' cell therapy
By partnering with ONK Therapeutics, Intellia joins CRISPR Therapeutics and Editas Medicine in striking deals that marry gene editing with a fast-emerging form of cancer immunotherapy.
By Ben Fidler • Feb. 15, 2022 -
Solskin via Getty Images
Sponsored by First Republic BankPrivate equity powers digital healthcare revolution
Read how private equity investment helps scale the push towards healthcare digitization.
By First Republic Bank • Feb. 7, 2022 -
Getty / Edited by BioPharma Dive
In England, Orchard secures rare agreement on gene therapy coverage
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.
By Ned Pagliarulo • Feb. 4, 2022 -
Courtesy of Sarepta
Sarepta reveals gene editing alliance with biotech exploring alternative delivery methods
The collaboration with GenEdit, a South San Francisco startup developing new ways of delivering gene editing tools into the body, dates back to December 2020, but has recently begun to advance.
By Ned Pagliarulo • Feb. 1, 2022 -
Sarah Silbiger via Getty Images
Bluebird faces longer wait for FDA verdict on experimental gene therapies
The FDA has extended by three months its review of Bluebird's treatments for beta thalassemia and a rare brain disorder, the latest delay in the company's lengthy road to market in the U.S.
By Ned Pagliarulo • Jan. 18, 2022 -
Danielle Ternes/BioPharma Dive
5 questions facing gene therapy in 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.
By Ned Pagliarulo • Jan. 13, 2022 -
Courtesy of Sarepta
Sarepta, with new Duchenne results, to broach 'possibility' of speedy gene therapy approval
The biotech will take new Phase 2 data to U.S. regulators, CEO Doug Ingram said Monday, though it still expects to need data from an ongoing late-stage trial to win approval.
By Ben Fidler • Jan. 10, 2022 -
cgtoolbox via Getty Images
Allogene cleared by FDA to resume 'off the shelf' cancer cell therapy trials
A "chromosomal abnormality" that led regulators to halt Allogene's trials was judged to be unrelated to its technology, a finding with important implications for the field of donor-derived cell therapies.
By Ben Fidler • Jan. 10, 2022 -
ismagilov via Getty Images
BioMarin plans return to FDA with updated data on hemophilia gene therapy
Two-year results from a Phase 3 study of Roctavian show treatment prevented bleeding, potentially giving BioMarin the data it needs to resubmit an application for approval.
By Ned Pagliarulo • Updated Jan. 10, 2022 -
Dan Kitwood via Getty Images
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 -
Artur Plawgo via Getty Images
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 -
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 -
Dr_Microbe via Getty Images
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 -
Artur Plawgo via Getty Images
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 -
Jeenah Moon via Getty Images
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 -
ismagilov via 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 -
ismagilov via 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 -
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 -
Stock via 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
Previous
