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

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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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

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

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

Researchers speed identification of DNA regions that regulate gene expression

Scienmag

Jude Children’s Research Hospital scientists have developed a highly efficient method to address a major challenge in biology–identifying the genetic ‘switches’ that regulate gene expression. Biology Gene Therapy Genes Genetics Medicine/Health

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

Bypassing broken genes

Scienmag

New approach to gene therapy can correct any disease-causing mutation within a gene UNIVERSITY PARK, Pa. — A new approach to gene editing using the CRISPR/Cas9 system bypasses disease-causing mutations in a gene, enabling treatment of genetic diseases linked to a single gene, such as cystic fibrosis, certain types of sickle cell anemia, and other […]. Medicine & Health Biology Cell Biology Gene Therapy Genetics Medicine/Health

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

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

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

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 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.

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

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

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

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

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

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

Lilly dives into gene editing

Bio Pharma Dive

Through a deal with Precision Biosciences, the pharma aims to develop therapies for genetic disorders, including Duchenne muscular dystrophy

Lilly gene-targeted drug shows potential for use across cancers

Bio Pharma Dive

Study results presented at AACR showed Retevmo shrank tumors in nine different cancers, building a case for the type of tissue-agnostic label that only three other medicines have secured

Gene 234

Could wider use of gene reserves protect rare species?

Scienmag

Credit: Melissa Minter, University of York UK landowners and conservationists welcome wider-spread use of Gene Conservation Units (GCUs) to help protect some of the rarest plants and insects, research at the University of York has shown.

Gene 62

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

Beam, capitalizing on stock surge, buys gene therapy tools developer for $120M

Bio Pharma Dive

Guide Therapeutics, the company Beam acquired, is working on ways to more efficiently deliver gene editing therapies into the body

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

Ultragenyx expands into Duchenne gene therapy with Solid Bio deal

Bio Pharma Dive

The rare disease drug developer is betting it can create a better DMD treatment by combining Solid's research with its manufacturing and gene delivery technology

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

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

Scientists use DNA origami to monitor CRISPR gene targeting

Scienmag

Technology and Engineering Biomechanics/Biophysics Biotechnology Chemistry/Physics/Materials Sciences Gene Therapy Genes Genetics

Editas, AbbVie rework gene editing deal as pioneering CRISPR trial resumes

Bio Pharma Dive

Development of EDIT-101, the first CRISPR-based therapy to be used in a company-led trial to alter genes within a person's body, will now move forward under Editas' sole ownership

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 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.

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

COVID-19: Scientists identify human genes that fight infection

Scienmag

– April 16, 2021 – Scientists at Sanford Burnham Prebys have identified a set of human genes that fight SARS-CoV-2 infection, the virus that causes COVID-19. Infectious Emerging Diseases Genes Genetics Immunology/Allergies/Asthma Infectious/Emerging Diseases Medicine/Health

Gene 62

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.

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

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

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.

Earthworms could help reduce antibiotic resistance genes in soil

Scienmag

Now, researchers reporting in ACS’ Environmental Science & Technology have dug up another possible role: reducing the number and relative abundance of antibiotic-resistance genes (ARGs) in soils from diverse ecosystems. Biology Agriculture Bacteriology Chemistry/Physics/Materials Sciences Entomology Genes Geology/Soil Infectious/Emerging DiseasesEarthworms improve the soil by aerating it, breaking down organic matter and mineralizing nutrients.

Gene 45

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