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

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

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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

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

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

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

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

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.

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

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

Biogen gene therapy misses goal in eye disease study

Bio Pharma Dive

The results are a setback for Biogen, which spent $800 million two years ago to acquire the treatment's developer, Nightstar Therapeutics

Gene therapy offers potential cure to children born without an immune system

Scienmag

Biology Clinical Trials Gene Therapy Health Care Immunology/Allergies/Asthma Medicine/Health Pediatrics

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

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.

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

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

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 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

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.

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

Preclinical done right: The importance of using relevant animal models in gene therapy studies

Bio Pharma Dive

Gene therapy is no longer an approach for the future. It's a technique used now

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

FDA wants more details on Sarepta gene therapy, potentially delaying final test

Bio Pharma Dive

A new request from the regulator could tighten the race between Sarepta and Pfizer to prove out a gene therapy for Duchenne muscular dystrophy

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

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

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

Gene therapy solution: The value of a CDMO as your end-to-end partner

Bio Pharma Dive

As gene therapy research continues to expand, innovators in this space will need CDMOs with highly specific expertise, facilities, and equipment

Gene therapy for placental insufficiency moves toward the clinic

Scienmag

publishers New Rochelle, NY, December 15, 2020–A new study identified an adenovirus gene therapy vector carrying a VEGF isoform. It can improve uterine blood flow in placental insufficiency, as reported in the peer-reviewed journal Human Gene Therapy.

Cancer case puts UniQure's hemophilia gene therapy on hold, raising alarms

Bio Pharma Dive

The FDA halted the AMT-061 program, which could be the first approved gene therapy for hemophilia B, because a patient who received the treatment appears to have developed liver cancer

Gene therapy offers a potential cure to children born without immune system

Scienmag

An international team of researchers at Great Ormond Street Hospital (GOSH), and University of California, Los Angeles (UCLA) have developed a gene therapy that successfully treated 48 out of 50 children with a form of severe combined immunodeficiency that leaves them without an immune system. Biology Clinical Trials Gene Therapy Genetics Immunology/Allergies/Asthma Medicine/Health Pediatrics

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

Solid Bio, eyeing comeback, points to 'totality' of new Duchenne gene therapy results

Bio Pharma Dive

The data keep Solid in the running in one of gene therapy's most competitive races, but still don't make clear whether the treatment is changing the trajectory of the disease

FDA gene therapy holdups suggest closer scrutiny by agency

Bio Pharma Dive

Requests for more data on manufacturing, clinical holds and a surprise rejection have led to questions of a tougher standard at the agency. Officials, however, point to a large and fast-advancing pipeline

Sarepta gene therapy misses goal in key muscular dystrophy study

Bio Pharma Dive

Company executives, however, blamed "improbably bad luck" for study enrollment that resulted in more older patients with milder disease receiving placebo, and pledged to press 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.

FDA INTERACT Meetings: Early Interactions for Cell and Gene Therapy Sponsors

Camargo

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers.

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.

Bluebird to withdraw gene therapy from Germany after dispute over price

Bio Pharma Dive

The biotech will cut staff, primarily in Europe, after negotiations with German health authorities resulted in a price lower than Bluebird had sought

2nd Gene Therapy for Neurological Disorders Europe

Pharma Phorum

Returning digitally in 2021, the 2 nd Annual Gene Therapy for Neurological Disorders Europe meeting will possibly be your last chance to attend a European meeting without the airmiles! The post 2nd Gene Therapy for Neurological Disorders Europe appeared first on.

First UK patient joins Pfizer’s phase 3 DMD gene therapy trial

Pharma Phorum

The first UK patient has enrolled on Pfizer’s phase 3 trial for its Duchenne muscular dystrophy (DMD) gene therapy. Participants will be followed in the CIFFREO study for five years after treatment with the investigational gene therapy.

Cellular benefits of gene therapy seen decades after treatment

Scienmag

An international collaboration between Great Ormond Street Hospital, the UCL GOS Institute for Child Health and Harvard Medical School has shown that the beneficial effects of gene therapy can be seen decades after the transplanted blood stem cells has been cleared by the body. Medicine & Health Biochemistry Cell Biology Gene Therapy Genetics Immunology/Allergies/Asthma Medicine/Health

AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases

Pharma Mirror

The post AavantiBio and Catalent Announce Partnership to Support Development and Manufacturing of Gene Therapies for Rare Genetic Diseases appeared first on Pharma Mirror Magazine. Pharmaceutical News AavantiBio Catalent Gene Therapies