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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 Therapy Related Topics 
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
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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
J&J pushes further into eye gene therapy
Bio Pharma Dive
DECEMBER 2, 2020
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
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
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
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.
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
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
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
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
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.
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
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
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
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
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.
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
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
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
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
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
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
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 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
Gene therapy for placental insufficiency moves toward the clinic
Scienmag
DECEMBER 15, 2020
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
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
Gene therapy offers a potential cure to children born without immune system
Scienmag
MAY 11, 2021
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
AUGUST 20, 2020
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
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
FDA gene therapy holdups suggest closer scrutiny by agency
Bio Pharma Dive
NOVEMBER 5, 2020
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
JANUARY 7, 2021
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
JANUARY 21, 2021
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
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.
Gene Therapy Medical Affairs
Pharma Phorum
NOVEMBER 20, 2020
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
APRIL 20, 2021
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
APRIL 19, 2021
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
MAY 13, 2021
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
MARCH 17, 2021
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
APRIL 28, 2021
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
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