article thumbnail

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

article thumbnail

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

Insiders

Sign Up for our Newsletter

This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

Prevail, Capsida partner to develop gene therapies for nervous system diseases

Bio Pharma Dive

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

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

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.

article thumbnail

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

article thumbnail

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

article thumbnail

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.

article thumbnail

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

article thumbnail

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

article thumbnail

Astellas makes another gene therapy play, takes stake in Taysha

Pharma Phorum

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.

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

‘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

article thumbnail

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

article thumbnail

CSL closes on EU approval for haemophilia B gene therapy

Pharma Phorum

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

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

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

article thumbnail

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.

article thumbnail

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

article thumbnail

Pfizer, Sangamo resume haemophilia gene therapy trial

Pharma Phorum

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.

article thumbnail

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

article thumbnail

Ferring wins FDA approval for bladder cancer gene therapy

Bio Pharma Dive

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

article thumbnail

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

article thumbnail

UniQure nabs another gene therapy for ALS

Bio Pharma Dive

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

article thumbnail

FDA approves first gene therapy for hemophilia B

Bio Pharma Dive

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

article thumbnail

FDA approves Adstiladrin as first gene therapy for NMIBC

Pharma Phorum

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.

article thumbnail

Cell One and CBM partner for cell and gene therapies

Pharmaceutical Technology

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.

article thumbnail

Sarepta, continuing its gene therapy push, helps launch a startup

Bio Pharma Dive

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

article thumbnail

Pfizer gene therapy research delayed by trial changes, safety questions

Bio Pharma Dive

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

article thumbnail

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.

article thumbnail

After setbacks, a gene therapy company mulls a sale

Bio Pharma Dive

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