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.

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 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

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

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

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

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

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

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

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

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

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

Biogen looks to build better gene therapies through latest deal

Bio Pharma Dive

The collaboration with Capsigen hands Biogen rights to a technology that could support the development of gene therapies for central nervous system and neuromuscular disorders

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

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

Bayer to buy AskBio for $2B in bid to become a gene therapy player

Bio Pharma Dive

Founded by gene therapy pioneer Jude Samulski, AskBio boasts a pipeline of experimental gene therapies as well as its own manufacturing capabilities

Gene Therapy for Muscular Disorders

Pharma Phorum

With clinical holds lifting there has been more investment and clinical progress in the gene therapy for muscular disorders space than ever before, yet the consequences of toxicity are not a distant memory. The post Gene Therapy for Muscular Disorders appeared first on.

With new results, Sarepta's 2nd gene therapy holds steady

Bio Pharma Dive

Stung by a clinical trial miss in Duchenne muscular dystrophy, the biotech now has promising, if early, data to back a gene therapy for a different rare neuromuscular disease

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

Gene therapy for sickle cell disease: progress and competition

Bio Pharma Dive

Treatments from Bluebird bio and CRISPR Therapeutics have shown considerable promise and could soon be nearing regulatory review. A full pipeline is growing behind them

Sarepta's Duchenne gene therapy clears study hurdle, although questions linger

Bio Pharma Dive

A version of the treatment made at commercial scale looks similar to what Sarepta used in early testing. But the findings are no longer the last step before an approval filing, as the company once hoped

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

How 2 scientific pioneers teamed up to run AskBio, Bayer's new gene therapy division

Bio Pharma Dive

Katherine High and Jude Samulski, two well-known gene therapy researchers, were longtime collaborators and competitors. Now they're colleagues at one of the world's largest drugmakers

New adaptable nanoparticle platform enables enhanced delivery of gene therapies

Scienmag

Credit: RCSI Scientists have developed polypeptide-based materials that act as effective vectors for delivering gene therapies. The first-of-its-kind platform enables the vectors to be adapted to suit the specific gene therapy cargo.

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

Innovative gene therapy ‘reprograms’ cells to reverse neurological deficiencies

Scienmag

Credit: The Ohio State University Wexner Medical Center A novel method of gene therapy is helping children born with a rare genetic disorder called AADC deficiency that causes severe physical and developmental disabilities. Biology Gene Therapy Medicine/Health Neurobiology Pediatrics Surgery

2nd Gene Therapy Immunogenicity

Pharma Phorum

The 2 nd Gene Therapy Immunogenicity Summit is the industry’s definitive forum enabling you to better modulate, measure and predict immune response to your gene therapy candidate. Performances of biosafety analytical assays commonly used in gene therapy development.

Innovation in cell and gene therapy: Insights from an industry specialist

Bio Pharma Dive

Cell and gene therapies (CGTs) are poised to revolutionize the landscape of biologic drugs that treat diseases and offer hope in areas where there was previously none

FDA lifts hold on UniQure gene therapy study after review of cancer case

Bio Pharma Dive

An investigation by UniQure determined the company's hemophilia gene therapy was "highly unlikely" to have caused a study volunteer's liver cancer, clearing the way for the FDA's green light

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

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

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

UniQure's gene therapy for hemophilia B meets first goal in key study

Bio Pharma Dive

The results are first to emerge from a late-stage test of a hemophilia B gene therapy, and show UniQure's treatment can restore clotting protein levels to mild or even near normal levels

NICE rejects Orchard’s gene therapy for rare childhood disease MLD

Pharma Phorum

Orchard Therapeutics’ gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance. Orchard has already offered a confidential discount on the therapy.

Biogen to spend $200M on gene therapy plant in North Carolina

Bio Pharma Dive

The planned factory is a strong sign of Biogen's interest in gene therapy, an area of research it has invested in through dealmaking over the past few years

FDA unexpectedly grounds a gene therapy for a rare heart disease

Bio Pharma Dive

Rocket Pharma’s Danon disease treatment — key to the company's quiet rise over the past year — is the latest gene therapy to be put on hold by the agency.

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

BioMarin resubmits haemophilia A gene therapy to the EMA

Pharma Times

Company initially withdrew a marketing authorisation application for the gene therapy last year

Gene Therapy Medical Affairs

Pharma Phorum

The inaugural Gene Therapy Medical Affairs Summit is dedicated to exploring the pivotal internal and external role that Medical Affairs plays in delivering gene therapies to patients. The post Gene Therapy Medical Affairs appeared first on.

Gene Therapy Comparability

Pharma Phorum

The inaugural Gene Therapy Comparability Summit is the only industry dedicated meeting to ensuring your manufacturing processes are effective and comparable at every stage of development, supporting a seamless transition to the larger scales required for commercialization.

Taysha Gene Therapies to Host Manufacturing Day

BioTech 365

Taysha Gene Therapies to Host Manufacturing Day Taysha Gene Therapies to Host Manufacturing Day Virtual event on Tuesday, July 27, 2021, at 10:00 a.m.

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

BioMarin's gene therapy rejection didn't shock everyone

Bio Pharma Dive

The FDA's decision not to approve Roctavian raises the question of why BioMarin was able to submit the hemophilia A therapy in the first place