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Addressing manufacturing challenges in gene therapy development

Bio Pharma Dive

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

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Voyager licenses gene therapy tools to Novartis

Bio Pharma Dive

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

The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo &rdquo

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5 questions facing gene therapy in 2022

Bio Pharma Dive

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

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FDA clears Intellia to start US tests of ‘in vivo’ gene editing drug

Bio Pharma Dive

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

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Astellas takes stake in gene therapy developer Taysha

Bio Pharma Dive

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

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5 questions facing gene therapy in 2023

Bio Pharma Dive

Buoyed by recent approvals, the field faces a pivotal year that’s likely to bring new treatments as well as more challenges

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A startup launches with plans to open up a gene and cell therapy bottleneck

Bio Pharma Dive

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

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Medicaid, with planned payment pilot, girds for influx of pricey gene therapies

Bio Pharma Dive

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

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5 questions facing gene therapy in 2021

Bio Pharma Dive

Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth

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As biotech retreats, gene therapy companies retrench and redraw plans

Bio Pharma Dive

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

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FDA sets out advice to developers of gene editing medicines

Bio Pharma Dive

Draft recommendations from the agency were generally straightforward, analysts said, although the document emphasized the potential safety risks of gene editing

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BioMarin's hemophilia gene therapy recommended for approval in Europe

Bio Pharma Dive

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

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At FDA meeting, gene therapy experts wrestle with field's blindspots

Bio Pharma Dive

A panel convened by the regulator suggested ways to make gene therapy research safer, but struggled to propose broader recommendations for the field

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AstraZeneca to acquire gene editing biotech LogicBio

Bio Pharma Dive

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

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Pfizer expands into gene editing with Beam research deal

Bio Pharma Dive

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

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Next-level quality control in cell and gene therapy

Bio Pharma Dive

Discover examples of how Droplet Digital™ PCR (ddPCR™) is enabling cell and gene therapies to reach further than ever before

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Gene editing: beyond the hype

Pharma Phorum

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.

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Sickle cell pipeline narrows as gene therapy developers rethink research plans

Bio Pharma Dive

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

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Bispecific cancer drugs and gene therapy advances: What to watch at next month’s ASH meeting

Bio Pharma Dive

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

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Roche digs deeper into gene therapy for the eye

Bio Pharma Dive

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

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Viral vector design for gene therapies

Bio Pharma Dive

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

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In England, Orchard secures rare agreement on gene therapy coverage

Bio Pharma Dive

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

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European regulators back hemophilia gene therapy

Bio Pharma Dive

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

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‘We have to find a way’: FDA seeks solutions to aid bespoke gene therapy

Bio Pharma Dive

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

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Gene therapy for Fabry: early stages, promising results

Bio Pharma Dive

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

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A high-profile gene therapy biotech takes aim at Huntington's

Bio Pharma Dive

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

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Orchard turns to layoffs in cutting gene therapy research

Bio Pharma Dive

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

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Yet another gene therapy developer turns to layoffs

Bio Pharma Dive

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

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Replay reveals second gene therapy spinout, debuting new biotech Telaria

Bio Pharma Dive

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

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Gene Therapy and Pharmacokinetics

Camargo

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.

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Vertex, Verve team up to develop a gene editing drug for liver disease

Bio Pharma Dive

The deal, which is worth $60 million upfront, broadens Vertex’s reach into gene editing and expands Verve’s research beyond heart disease

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Astellas, despite recent setbacks, opens $100M gene therapy plant

Bio Pharma Dive

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

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FDA lifts hold on Astellas’ Pompe gene therapy

Pharma Phorum

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.

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Cyagen and Neurophth partner to develop gene therapy vectors

Pharmaceutical Technology

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.

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Vertex aligns with another CRISPR gene editing startup

Bio Pharma Dive

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

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PTC's first gene therapy gains European regulator's backing

Bio Pharma Dive

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

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Gene therapy for hemophilia: So close, yet so far away

Bio Pharma Dive

Significant setbacks for pace-setting programs from BioMarin and UniQure have renewed questions about gene therapy’s promise, and its safety

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Scientists home in on cause of Duchenne gene therapy side effect

Bio Pharma Dive

An unusual collaboration among gene therapy developers suggests certain mutations could be behind "peculiar" side effects experienced by several patients treated in clinical trials

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Astellas licenses companion drug for Pompe gene therapy

Pharma Phorum

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.

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Roche writes off $3B on lower sales forecasts for gene therapies, cancer drugs

Bio Pharma Dive

Among the drugs Roche recorded impairment charges on are four gene therapies acquired in the company's 2019 deal for Spark Therapeutics

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Record funding flowed into cell, gene therapy companies last year

Bio Pharma Dive

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

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Takeda takes aim at a biotech's gene therapy work

Bio Pharma Dive

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