Addressing manufacturing challenges in gene therapy development
Bio Pharma Dive
MARCH 20, 2023
As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures
Gene Therapy Related Topics 
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Voyager licenses gene therapy tools to Novartis
Bio Pharma Dive
MARCH 6, 2023
The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures
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5 questions facing gene therapy in 2022
Bio Pharma Dive
JANUARY 13, 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
5 questions facing gene therapy in 2023
Bio Pharma Dive
JANUARY 12, 2023
Buoyed by recent approvals, the field faces a pivotal year that’s likely to bring new treatments as well as more challenges
Astellas takes stake in gene therapy developer Taysha
Bio Pharma Dive
OCTOBER 24, 2022
Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders
Prevail, Capsida partner to develop gene therapies for nervous system diseases
Bio Pharma Dive
JANUARY 4, 2023
The Lilly-owned subsidiary will pay $55 million to gain access to Capsida’s AAV gene therapy technology in a deal aimed at central nervous system disorders
Medicaid, with planned payment pilot, girds for influx of pricey gene therapies
Bio Pharma Dive
FEBRUARY 21, 2023
The proposed model could help state Medicaid agencies explore different kinds of outcomes-based payment schemes, but may come too late to prepare for the first sickle cell gene therapy
As biotech retreats, gene therapy companies retrench and redraw plans
Bio Pharma Dive
MARCH 24, 2022
At least nine biotechs working in cell or gene therapy have announced layoffs, cost cuts or restructured their research since December — restructurings that have coincided with a stock market downturn
BioMarin's hemophilia gene therapy recommended for approval in Europe
Bio Pharma Dive
JUNE 24, 2022
The decision brings what could be the first approved hemophilia gene therapy, Roctavian, closer to market, after a series of regulatory setbacks that have delayed its arrival
At FDA meeting, gene therapy experts wrestle with field's blindspots
Bio Pharma Dive
SEPTEMBER 3, 2021
A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field
Next-level quality control in cell and gene therapy
Bio Pharma Dive
JULY 25, 2022
Discover examples of how Droplet Digital™ PCR (ddPCR™) is enabling cell and gene therapies to reach further than ever before
European regulators back hemophilia gene therapy
Bio Pharma Dive
DECEMBER 16, 2022
The EMA’s drug review committee recommended five new drugs be approved in Europe during its December meeting, including CSL’s Hemgenix, AstraZeneca’s Imjudo and Amicus’ Pombiliti
FDA lifts hold on Astellas’ Pompe gene therapy
Pharma Phorum
JANUARY 20, 2023
The FDA has delivered some good news to Astellas’ troubled gene therapy unit, lifting a clinical hold on its Pompe disease candidate AT845 and clearing the way for dosing of patients to resume. The post FDA lifts hold on Astellas’ Pompe gene therapy appeared first on.
Sickle cell pipeline narrows as gene therapy developers rethink research plans
Bio Pharma Dive
FEBRUARY 23, 2023
Graphite Bio and Sangamo are stopping work on their respective sickle cell gene therapies, while Intellia revealed partner Novartis ended development of its genetic treatment for the blood disease
Viral vector design for gene therapies
Bio Pharma Dive
APRIL 4, 2022
Safety issues in clinical trials cast doubt on the ability of gene therapy to safely mitigate disease, prompting further research into understanding the virus-host interaction that resulted in new generations of viral vectors
Astellas licenses companion drug for Pompe gene therapy
Pharma Phorum
JANUARY 10, 2023
Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene therapy for inherited neuromuscular disorder Pompe disease. The post Astellas licenses companion drug for Pompe gene therapy appeared first on.
Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting
Bio Pharma Dive
NOVEMBER 4, 2022
Competition is increasing behind newly approved drugs from Roche and J&J, while the long-term benefit of gene therapies for chronic blood disorders is being put to the test
Roche digs deeper into gene therapy for the eye
Bio Pharma Dive
JULY 20, 2022
The Swiss drugmaker, which already owns rights to a marketed gene therapy for inherited vision loss, will work with startup Avista Therapeutics to develop better delivery tools for the complex treatments
Astellas makes another gene therapy play, takes stake in Taysha
Pharma Phorum
OCTOBER 25, 2022
Astellas has been having some safety-related issues with its gene therapy programmes of late, but it shows no signs of lessened enthusiasm for the category – as a new alliance with US biotech Taysha demonstrates.
In England, Orchard secures rare agreement on gene therapy coverage
Bio Pharma Dive
FEBRUARY 4, 2022
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
Gene therapy for Fabry: early stages, promising results
Bio Pharma Dive
JULY 22, 2021
Three companies are testing their gene therapies in early clinical trials, with initial results due later this year. Others are in preclinical stages and aim to follow soon
A high-profile gene therapy biotech takes aim at Huntington's
Bio Pharma Dive
DECEMBER 8, 2021
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
‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy
Bio Pharma Dive
OCTOBER 13, 2022
Gene therapies could help treat many ultra-rare diseases. But they may not get developed if drugmakers can’t build a sustainable business around them, CBER director Peter Marks said at a conference
Orchard turns to layoffs in cutting gene therapy research
Bio Pharma Dive
MARCH 30, 2022
The biotech company plans to reduce its workforce by 30%, or by about 65 employees, in the latest example of a gene therapy developer restructuring
CSL closes on EU approval for haemophilia B gene therapy
Pharma Phorum
DECEMBER 16, 2022
CSL’s gene therapy for haemophilia B has been recommended for approval by the EMA’s human medicine committee, setting up a decision by the European Commission early next year. CSL launched the therapy in the US with a list price of $3.5
Yet another gene therapy developer turns to layoffs
Bio Pharma Dive
APRIL 1, 2022
Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines
Replay reveals second gene therapy spinout, debuting new biotech Telaria
Bio Pharma Dive
NOVEMBER 21, 2022
Telaria is the second spinout in Replay’s “hub-and-spoke” model, and will focus on developing a gene therapy for recessive dystrophic epidermolysis bullosa
Astellas, despite recent setbacks, opens $100M gene therapy plant
Bio Pharma Dive
JUNE 8, 2022
The opening of the plant, which could create more than 200 new jobs, comes on the heels of several significant issues with the company’s most advanced gene therapy program
PTC's first gene therapy gains European regulator's backing
Bio Pharma Dive
MAY 20, 2022
The brain-infused treatment for a rare neurological disease would be one of only three gene therapies commercially available in the EU. PTC plans to ask for FDA approval later this year
Cyagen and Neurophth partner to develop gene therapy vectors
Pharmaceutical Technology
NOVEMBER 8, 2022
Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Scientists home in on cause of Duchenne gene therapy side effect
Bio Pharma Dive
MAY 17, 2022
An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials
Pfizer, Sangamo resume haemophilia gene therapy trial
Pharma Phorum
SEPTEMBER 26, 2022
Pfizer and Sangamo have restarted recruitment of patients into their registration trial for haemophilia A gene therapy giroctocogene fitelparvovec, after the FDA lifted a clinical hold on the study. The post Pfizer, Sangamo resume haemophilia gene therapy trial appeared first on.
Takeda takes aim at a biotech's gene therapy work
Bio Pharma Dive
OCTOBER 12, 2021
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
Ferring wins FDA approval for bladder cancer gene therapy
Bio Pharma Dive
DECEMBER 19, 2022
The OK caps a long development journey for the treatment, which was previously turned back by the agency. Ferring doesn’t expect it to be available until the second half of next year, however
Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs
Bio Pharma Dive
FEBRUARY 3, 2023
Among the drugs Roche recorded impairment charges on are four gene therapies acquired in the company's 2019 deal for Spark Therapeutics
UniQure nabs another gene therapy for ALS
Bio Pharma Dive
JANUARY 31, 2023
For $10 million up front, UniQure has licensed rights to its second experimental treatment for the condition, a medicine from startup Apic Bio that should enter human testing later this year
FDA approves first gene therapy for hemophilia B
Bio Pharma Dive
NOVEMBER 22, 2022
The treatment, which is for the less common “B” form of the bleeding disorder, will be sold in the U.S. by maker CSL for $3.5 million
FDA approves Adstiladrin as first gene therapy for NMIBC
Pharma Phorum
DECEMBER 19, 2022
Ferring Pharma ’s Adstiladrin is a non-replicating (cannot multiply in human cells) adenoviral vector-based gene therapy. The application was granted Priority Review, Breakthrough Therapy, and Fast Track designations.
Cell One and CBM partner for cell and gene therapies
Pharmaceutical Technology
MARCH 10, 2023
Cell One Partners has announced a strategic collaboration with the Center for Breakthrough Medicines (CBM) to expedite the development and commercialisation of cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Sarepta, continuing its gene therapy push, helps launch a startup
Bio Pharma Dive
OCTOBER 22, 2020
The biotech has joined a group of investors to form a new gene therapy developer led by one of its longtime executives and backed with $107 million
Pfizer gene therapy research delayed by trial changes, safety questions
Bio Pharma Dive
NOVEMBER 3, 2021
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
Gene Therapy and Pharmacokinetics
Camargo
JULY 27, 2021
How and When to Incorporate PK Design into Your Gene Therapy Development Plan. Gene therapy, which was in its infancy around 30 years ago, is now becoming a more prominent treatment method in many therapeutic areas, from personalized therapy to mass vaccinations against COVID-19.
After setbacks, a gene therapy company mulls a sale
Bio Pharma Dive
APRIL 28, 2022
Sio Gene Therapies, formerly known as Axovant, said it's focused on conserving cash, and has decided to significantly reduce its workforce and terminate licensing agreements on its last two pipeline programs
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