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 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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Moderna inks another gene editing deal
Bio Pharma Dive
FEBRUARY 22, 2023
The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo &rdquo
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
FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug
Bio Pharma Dive
MARCH 2, 2023
The announcement, which follows recent regulatory setbacks for some of Intellia’s peers, is a “big win” for the gene editing field, according to one analyst
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
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
A startup launches with plans to open up a gene and cell therapy bottleneck
Bio Pharma Dive
JANUARY 31, 2023
The pipeline of CAR-T therapies and ex vivo gene therapies has swelled in recent years, but manufacturing hasn’t been able to keep up with demand
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
5 questions facing gene therapy in 2021
Bio Pharma Dive
JANUARY 8, 2021
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth
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
FDA sets out advice to developers of gene editing medicines
Bio Pharma Dive
MARCH 16, 2022
Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing
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
AstraZeneca to acquire gene editing biotech LogicBio
Bio Pharma Dive
OCTOBER 3, 2022
Shares in LogicBio had lost nearly all their value since a 2018 IPO, bringing the company to the brink of Nasdaq delisting. The buyout deal represents a premium of over 600
Pfizer expands into gene editing with Beam research deal
Bio Pharma Dive
JANUARY 10, 2022
Pfizer's work with mRNA vaccines led it explore other applications of the technology, resulting in a multi-year partnership with the high-profile biotech Beam Therapeutics on gene editing treatments for rare diseases
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
Gene editing: beyond the hype
Pharma Phorum
JANUARY 29, 2021
Cutting edge’ is, for once, a truly apt description when it comes to gene editing – both because the field is pushing medicine into areas we might never have dreamed possible, and because these technologies involve literally cutting DNA at a specific point in the genome.
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
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
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
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
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
‘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
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
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
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
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.
Vertex, Verve team up to develop a gene editing drug for liver disease
Bio Pharma Dive
JULY 20, 2022
The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease
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
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.
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.
Vertex aligns with another CRISPR gene editing startup
Bio Pharma Dive
OCTOBER 26, 2021
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
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
Gene therapy for hemophilia: So close, yet so far away
Bio Pharma Dive
JANUARY 28, 2021
Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety
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
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.
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
Record funding flowed into cell, gene therapy companies last year
Bio Pharma Dive
MARCH 16, 2021
Nearly $20 billion was invested in biotechs developing cell-, gene- and tissue-based treatments in 2020, according to a new report from industry group ARM
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
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