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5 questions facing gene therapy in 2021
Bio Pharma Dive
JANUARY 8, 2021
Gene therapy developers will need to address a series of setbacks that have shaded optimism in the sector's fast growth
Gene Related Topics 
Gene therapy for hemophilia: So close, yet so far away
Bio Pharma Dive
JANUARY 28, 2021
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
MARCH 16, 2021
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
OCTOBER 22, 2020
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
MAY 11, 2021
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
MAY 6, 2021
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
APRIL 1, 2021
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
MARCH 11, 2021
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
APRIL 21, 2021
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
APRIL 28, 2021
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
NOVEMBER 5, 2020
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
FEBRUARY 25, 2021
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
APRIL 13, 2021
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
MAY 11, 2021
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
JANUARY 29, 2021
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
JANUARY 21, 2021
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
MARCH 19, 2021
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
NOVEMBER 19, 2020
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
NOVEMBER 5, 2020
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
APRIL 26, 2021
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
OCTOBER 26, 2020
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
MAY 14, 2021
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
NOVEMBER 20, 2020
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
APRIL 11, 2021
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
Could wider use of gene reserves protect rare species?
Scienmag
MAY 9, 2021
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.
Preclinical done right: The importance of using relevant animal models in gene therapy studies
Bio Pharma Dive
OCTOBER 1, 2020
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
FEBRUARY 23, 2021
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
SEPTEMBER 10, 2020
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
OCTOBER 23, 2020
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
MARCH 4, 2021
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
OCTOBER 30, 2020
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
FEBRUARY 23, 2021
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
AUGUST 7, 2020
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
OCTOBER 14, 2020
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
JANUARY 21, 2021
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
SEPTEMBER 29, 2020
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
– 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
2nd Gene Therapy Immunogenicity
Pharma Phorum
APRIL 16, 2021
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
MAY 11, 2021
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
DECEMBER 21, 2020
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
MAY 2, 2021
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
MAY 12, 2021
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.
Solid Bio, eyeing comeback, points to 'totality' of new Duchenne gene therapy results
Bio Pharma Dive
MARCH 16, 2021
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
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