Gene Therapy: Page 10
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UK biotech AlveoGene launches with plans for inhaled gene therapy
The company is working on a treatment of alpha-1 antitrypsin deficiency, which it claims it can deliver direct to lung cells via a nebulizer.
By Kristin Jensen • Sept. 14, 2023 -
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Wave readies clinical testing for first RNA editing therapy
The biotech, one of several advancing RNA editing medicines, expects to obtain “proof-of-mechanism” data in patients with the inherited disorder alpha-1 antitrypsin deficiency by next year.
By Kristin Jensen • Updated Sept. 5, 2023 -
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 -
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Beam begins US testing of first-of-its-kind ‘base editing’ cancer drug
The announcement marks progress for Beam after trial delays, and is the first time in the U.S. that a patient has received a base editing treatment.
By Ben Fidler • Sept. 5, 2023 -
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Startup Cellares adds $255M as investors pour cash into cell therapy production
Bristol Myers Squibb is among those backing the company, which claims the manufacturing capacity at its New Jersey plant can surpass that of conventional CDMO facilities.
By Kristin Jensen • Aug. 24, 2023 -
Permission granted by MilliporeSigma
Sponsored by MilliporeSigma5 ways experience matters in gene therapy manufacturing
By partnering with an accomplished CTDMO, gene therapy innovators can benefit from guidance and support.
Aug. 21, 2023 -
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Bruker to buy PhenomeX and its cell research tools for $108M
The company said PhenomeX’s technology will help its customers speed development and manufacturing of cell and gene therapies.
By Susan Kelly • Aug. 18, 2023 -
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FDA won’t hold advisory meeting for Bluebird’s sickle cell gene therapy
The decision stands in contrast to the agency’s plans for a would-be rival gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that is also under a regulatory review.
By Kristin Jensen • Aug. 16, 2023 -
Peddalanka Ramesh Babu via Getty Images
Precision sells lymphoma drug to Imugene in retreat from cell therapy research
The struggling biotech also plans to offload other cell therapy assets, including a multiple myeloma therapy, and focus exclusively on gene editing medicines.
By Ben Fidler • Aug. 16, 2023 -
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HIV cell therapy startup Addimmune heads to Wall Street via blank-check merger
The deal will help fund a cell-based treatment that completed Phase 1 testing last year and is designed to provide a “functional cure” for patients with HIV infections.
By Ben Fidler • Aug. 10, 2023 -
Courtesy of Regeneron Pharmaceuticals
Regeneron ups genetic medicine investment with Decibel buyout
Third Rock-founded Decibel, which has shed much of its value since a 2021 IPO, expected to run out of money within a year.
By Jonathan Gardner • Aug. 9, 2023 -
Courtesy of Sarepta
Sarepta offers early look at closely watched gene therapy launch
One patient received its Duchenne gene therapy, Elevidys, this week and a handful more could follow shortly, surprising some analysts and raising near-term sales expectations.
By Ben Fidler • Aug. 3, 2023 -
Permission granted by Intergalactic Therapeutics
A once buzzy gene therapy startup lays off staff and shuts down
Intergalactic, a developer of non-viral gene therapies built by ex-Biogen executive Michael Ehlers, is folding less than two years after its launch.
By Kristin Jensen • Aug. 2, 2023 -
Permission granted by Vertex Pharmaceuticals
Vertex taking ‘flexible’ approach to payment talks for experimental CRISPR therapy
The company is discussing various reimbursement models for its sickle cell treatment with government and commercial insurers ahead of an FDA approval decision later this year.
By Ned Pagliarulo • Aug. 2, 2023 -
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AstraZeneca buys Pfizer’s early gene therapy work for up to $1B
The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.
By Ben Fidler • July 28, 2023 -
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Kincell spins out of cell therapy biotech, joining wave of manufacturing startups
The company is the latest to emerge with plans to make production of cell-based medicines easier for researchers and biotechs.
By Ben Fidler • July 26, 2023 -
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Gene therapy biotech Kriya adds $150M in fresh funds
Led by Vivek Ramaswamy's brother, Kriya has now raised over $600 million to fund gene therapy research spanning ophthalmology, neurology and metabolic disease.
By Kristin Jensen • July 26, 2023 -
Courtesy of Editas Medicine
Editas names Biogen veteran as top scientist in latest executive change
Linda Burkly’s appointment follows the exit of CSO Mark Shearman, whose departure Editas announced alongside layoffs earlier this year.
By Gwendolyn Wu • July 24, 2023 -
Permission granted by Center for Breakthrough Medicines
Sponsored by Center for Breakthrough MedicinesCell and gene therapy is coming of age
Outsourcing in the cell and gene therapy industry is booming and no longer considered just a “trend.”
July 24, 2023 -
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A cancer biotech’s collapse gives Neurogene a path to Wall Street
The startup, a developer of gene therapies for brain diseases like Rett syndrome, will bypass an IPO by merging with struggling drugmaker Neoleukin.
By Delilah Alvarado • July 18, 2023 -
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Avrobio halts gene therapy research and considers a sale
The decision to explore “strategic alternatives” comes about six weeks after the company sold its most advanced treatment to Novartis for nearly $90 million.
By Ned Pagliarulo • July 12, 2023 -
Permission granted by Jennifer Arcure
Astellas buys rights to 4D Molecular’s technology in latest gene therapy deal
The licensing agreement for the biotech’s viral vector platform follows recent Astellas agreements with Taysha Gene Therapies and Kate Therapeutics.
By Delilah Alvarado • July 10, 2023 -
Permission granted by Center for Breakthrough Medicine
Sponsored by Center for Breakthrough MedicinesOvercoming AAV manufacturing challenges
Overcome issues with AAV vector scalability and reproducibility. Enable a wider application of gene therapies in a cost-effective and sustainable way.
July 10, 2023 -
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Pfizer backs CRISPR biotech Caribou in latest cell therapy investment
Five years after helping launch Allogene Therapeutics, Pfizer is backing another developer of “off-the-shelf” cell therapies in a deal that gives the drugmaker the chance to acquire a multiple myeloma treatment.
By Kristin Jensen • July 6, 2023 -
Courtesy of Sarepta
Sarepta sells FDA fast pass earned from Duchenne gene therapy approval
The deal enables Sarepta to quickly cash in on the clearance of Elevidys, with $102 million in new funds to help bankroll its drug research.
By Jonathan Gardner • July 5, 2023 -
Courtesy of BioMarin Pharmaceutical
With FDA approval in hand, BioMarin lays out plan to sell $2.9M gene therapy
The agency granted a long-awaited clearance last Thursday, but unexpected aspects of the hemophilia treatment’s label had some investors worried about its commercial prospects.
By Jacob Bell • June 30, 2023
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