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

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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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

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

Modernizing Pharmaceutical Supply Chains

Speaker: Nimesh Patel, Gregory Demitrack, and Sarah Barnes Humphrey

Register for our upcoming webinar to better understand the state of the pharmaceutical supply chain. FourKites partnered with Accenture to survey supply chain executives working in pharmaceuticals, biotech, cell and gene therapy, medtech, and other related industries.

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

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

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

Avrobio stops work on rare disease gene therapy after unexpected study results

Bio Pharma Dive

The biotech's treatment was one of the furthest along in testing for Fabry, a rare inherited disease that's become a target for gene therapy developers

Biogen gene therapy deal has yet to bear fruit

Bio Pharma Dive

Another gene therapy from Nightstar Therapeutics, which Biogen bought for $800 million in 2019, has failed in a key clinical study

J&J pushes further into eye gene therapy

Bio Pharma Dive

The pharma licensed a treatment for a severe type of age-related vision loss, building on past deals that gave it a foothold in gene therapy

Years later, a first-of-its-kind treatment shows the power, and limits, of gene therapy

Bio Pharma Dive

Luxturna, approved four years ago as the first gene therapy for an inherited disease in the U.S., is improving sight and quality of life for several of the patients who received it

Intergalactic Therapeutics launches with $75M to build a new type of gene therapy

Bio Pharma Dive

Founded by former Biogen executive Michael Ehlers and backed by Apple Tree Partners, Intergalactic aims to create non-viral gene therapies delivered via an advanced form of electroporation

Bluebird's next gene therapy gets backing from European regulator

Bio Pharma Dive

The treatment, for a progressive, often deadly brain disease, could soon become Bluebird's third approved product and one of only a handful of marketed gene therapies in the world

What to expect at the FDA's two-day meeting on gene therapy safety

Bio Pharma Dive

A group of advisers convened by the regulator will spend Thursday and Friday weighing a range of safety worries with gene therapy, and how best to prevent them

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

Audentes CEO exits as Astellas rebrands a gene therapy buyout

Bio Pharma Dive

Natalie Holles, who was appointed CEO of Audentes soon after Astellas acquired the gene therapy developer in 2019, has departed amid the company's integration

FDA halts tests of BioMarin drug amid heightened focus on gene therapy safety

Bio Pharma Dive

On the heels of a two-day FDA meeting in which experts debated the risks of gene therapy, the biotech said its early-stage PKU treatment was associated with cancer in mice

Bluebird hit with another hold for sickle cell gene therapy

Bio Pharma Dive

Study enrollment and treatment of sickle cell patients under 18 will be paused for safety reasons, another hurdle in a series of setbacks for the company

Bluebird, winding down in Europe, withdraws another rare disease gene therapy

Bio Pharma Dive

The biotech won EU approval for two gene therapies, Zynteglo and Syksona, both of which it's now said it will pull from market after difficulties negotiating reimbursement

A biotech's eye gene therapy faces longer odds as serious side effects pile up

Bio Pharma Dive

Five trial participants with diabetic macular edema who received Adverum Biotechnologies' treatment experienced side effects never before reported for an eye gene therapy

Boehringer Ingelheim takes next step in development of cystic fibrosis gene therapy

Bio Pharma Dive

Gene therapy could be a solution for the remaining 10%, but the path forward is challenging Drugs from Vertex can effectively treat the lung disease in as much as 90% of patients.

The 'dovetail' partnership meant to turn Boston Children's into a hub for gene therapy startups

Bio Pharma Dive

A five-year alliance with high-profile biotech ElevateBio is designed to get around a bottleneck that has held back the institution’s gene therapy work

Gene therapy for Duchenne muscular dystrophy: Nearing final tests

Bio Pharma Dive

Sarepta and Pfizer are advancing their experimental treatments into late-stage trials, while others are accelerating research in what's become one of gene therapy's most active fields

Duchenne patient dies in Pfizer gene therapy study

Bio Pharma Dive

The tragic news follows changes Pfizer recently made to the design of another study testing the therapy due to side effects seen in some participants

A biotech startup raises $117M to deliver gene therapies in a new way

Bio Pharma Dive

Flagship Pioneering, the biotech incubator that created Moderna, is helping fund Ring Therapeutics, a startup hoping to disrupt gene therapy development through the use of a different viral vector

Amicus, looking for a boost, sells its gene therapy work to a SPAC

Bio Pharma Dive

The value of the biotech's growing gene therapy portfolio has been "unrecognized" by investors, said CEO John Crowley, leading to a sudden shift for an initiative that began just three years ago

PDUFA VII: Implications for Developers of Cell and Gene Therapies

Camargo

Many of the advancements included in PDUFA VII are driven in large part by an increase in the development of innovative products such as cell and gene therapies. The increase is designed to strengthen CBER’s capacity and capabilities for regulating cell and gene therapies.

Axovant shares sink on manufacturing delay for Parkinson's gene therapy

Bio Pharma Dive

A longer-than-expected timeline for developing a new manufacturing process means a key study for Axovant's top gene therapy likely won't begin until 2022

Bluebird faces delay for sickle cell gene therapy as rivals inch closer

Bio Pharma Dive

The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers

Bluebird, with little fanfare, is first to bring a second gene therapy to market

Bio Pharma Dive

The approval of Skysona for a genetic brain disease is a milestone for one of gene therapy's pioneering companies, but isn't expected to turn Bluebird's financial fortunes around

Former AveXis executives launch gene therapy startup with uncommon targets

Bio Pharma Dive

Sean Nolan and Joe Nolan, former leaders at the Zolgensma developer, are behind Jaguar Gene Therapy, which plans to research treatments for a rare metabolic disease as well as genetically linked autism and diabetes

How Rocket Pharma quietly became one of gene therapy's high flyers

Bio Pharma Dive

An unconventional strategy is behind the rise of Rocket, a New Jersey biotech whose executives boldly talk of turning into the "Genentech of gene therapy

Bluebird resumes marketing gene therapy in Europe

Bio Pharma Dive

Zynteglo sales have been on hold since February, when a patient in a clinical trial of another, related Bluebird medicine developed leukemia

Cell and Gene Therapy Bioassay – A Prerequisite for the ATMPs Development

Roots Analysis

Bioassay – A Prerequisite for the Cell and Gene Therapy Development. Over the past few years, investigational new drug (IND) filings for cell and gene therapy product have significantly increased. Companies Offering Bioassay Services for Cell and Gene Therapies.

Sarepta outlines final push for Duchenne gene therapy

Bio Pharma Dive

The biotech still believes a speedy approval filing for the closely watched treatment is possible, but expects to have to wait for the results of the recently launched Phase 3 trial, CEO Doug Ingram said on a conference call

Fourth trial volunteer dies in Astellas gene therapy study

Bio Pharma Dive

The study, which was suspended following three deaths last year, had been restarted in February after Astellas lowered the treatment dose used

BioMarin partners with Allen Institute to develop gene therapies for the brain

Bio Pharma Dive

Far along in testing for a hemophilia treatment, BioMarin is expanding its gene therapy research into diseases of the central nervous system and aims to use some of the institute's technology

Lilly, Arch join Deerfield in backing new gene therapy biotech Jaguar

Bio Pharma Dive

Just two months after launching, Jaguar Gene Therapy has raised another $139 million for a preclinical pipeline aimed at gene-linked autism and diabetes

NIH, FDA spearhead broad partnership to speed gene therapy research

Bio Pharma Dive

The two institutions will work with drugmakers and non-profit groups on a wide-ranging initiative meant to make the complex treatments easier and less costly produce

Roche, aiming to make better gene therapies, turns to an emerging startup

Bio Pharma Dive

The Swiss pharma is the third large drugmaker to partner with Harvard spinout Dyno Therapeutics, a small biotech aiming to improve gene therapy technology

Bluebird faces longer wait for FDA verdict on experimental gene therapies

Bio Pharma Dive

The FDA has extended by three months its review of Bluebird's treatments for beta thalassemia and a rare brain disorder, the latest delay in the company's lengthy road to market in the U.S.

BioMarin plans return to FDA with updated data on hemophilia gene therapy

Bio Pharma Dive

Two-year results from a Phase 3 study of Roctavian show treatment prevented bleeding, potentially giving BioMarin the data it needs to resubmit an application for approval