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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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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
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
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
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
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 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
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
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
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
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
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
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
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.
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.
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
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
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
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
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
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.
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
Taysha Gene Therapies Announces New cGMP Gene Therapy Manufacturing Facility
BioTech 365
DECEMBER 17, 2020
Scientists develop new gene therapy for eye disease
Scienmag
NOVEMBER 25, 2020
Medicine & Health Gene Therapy Genes Genetics Medicine/Health Ophthalmology
Pfizer beats Sarepta to the start of first late-stage Duchenne gene therapy trial
Bio Pharma Dive
JANUARY 7, 2021
The first patient enrolled in a Phase 3 trial of Pfizer's treatment was given the experimental one-time therapy on Dec.
bluebird bio ‘baffled’ after NICE rejects beta-thalassaemia gene therapy
Pharma Phorum
FEBRUARY 16, 2021
It’s back to the drawing board for bluebird bio and its discussions with NICE, which has rejected its beta thalassaemia gene therapy Zynteglo for regular NHS use in first draft guidance. Market Access News beta thalassaemia bluebird bio gene therapy Haematology NICE Zynteglo
BioMarin tries to get hemophilia gene therapy back on track with positive data
Bio Pharma Dive
JANUARY 11, 2021
Full one-year results from a Phase 3 study show the therapy, known as Roctavian, sharply reduced bleeds and use of blood-clotting treatments
Riding the Gene Therapy Wave, Biogen Releases Plans for Facility
BioSpace
MARCH 3, 2021
Biogen plans to build a new gene therapy manufacturing plant in Research Triangle Park, North Carolina. The site is on the company’s manufacturing campus
A gene therapy pipeline takes shape for a cluster of rare diseases
Bio Pharma Dive
FEBRUARY 12, 2021
Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots
Are Gene Therapies the Medicine of the Future?
BioSpace
JANUARY 25, 2021
Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks
Neurocine ends partnership with Voyager for Parkinson's gene therapy
Bio Pharma Dive
FEBRUARY 3, 2021
The loss of Neurocrine as a collaborator is another blow to Voyager after the FDA placed a clinical hold on the experimental treatment
Bluebird reveals new details on cancer cases in gene therapy trial
Bio Pharma Dive
FEBRUARY 26, 2021
Executives shared an update on the biotech's investigation of the two cases at an investor conference, rather than via a statement or regulatory filing
Catalent to invest $130M in gene therapy plant near Baltimore airport
Bio Pharma Dive
SEPTEMBER 10, 2020
The contract manufacturer is riding a boom in demand for both its gene therapy and vaccine manufacturing services
Bluebird stops studies of sickle cell gene therapy after new cancer cases
Bio Pharma Dive
FEBRUARY 16, 2021
The biotech is also suspending sales of its related treatment Zynteglo following a leukemia diagnosis in a clinical trial volunteer and a case of a cancer-like bone marrow disease in another
Pfizer invests in Homology and adds to its gene therapy ambitions
Bio Pharma Dive
NOVEMBER 10, 2020
Along with an equity investment, Pfizer will get first dibs on any potential transaction involving Homology's lead programs, which the biotech is developing for a rare metabolic disorder
Biogen pushes further into gene therapy for the eye with new deal
Bio Pharma Dive
JANUARY 6, 2021
Partnering with German biotech ViGeneron bolsters Biogen's pipeline of drugs for eye diseases, which includes assets acquired through its buyout of Nightstar Therapeutics
2nd Gene Therapy for Blood Disorders
Pharma Phorum
NOVEMBER 20, 2020
2020 has been an exciting year for gene therapies targeting blood disorders. With pipelines advancing and emerging companies joining the blood disorders field, it is essential for gene therapy developers to communicate and share lessons learned to accelerate their programs to market.
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
Bayer buys cell & gene therapy firm AskBio for $4 billion
Pharma Phorum
OCTOBER 26, 2020
Bayer is making clear its ambitions in cell and gene therapy with a $4 billion acquisition of US biotech Asklepios BioPharmaceutical, also known as AskBio. The post Bayer buys cell & gene therapy firm AskBio for $4 billion appeared first on.
Taysha Gene Therapies Announces Support of Rare Disease Day 2021
BioTech 365
FEBRUARY 26, 2021
Taysha Gene Therapies Announces Support of Rare Disease Day 2021 Taysha Gene Therapies Announces Support of Rare Disease Day 2021 Launches #RareAlly initiative to recognize those in the rare disease community who inspire and motivate us each day Furthers efforts … Continue reading → Business Wire
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
Gene therapy strategy found effective in mouse model of hereditary disease TSC
Scienmag
JANUARY 8, 2021
Biology Gene Therapy Medicine/Health NeurobiologyTreatment reverses aspects of tuberous sclerosis complex, which is characterized by noncancerous tumors in multiple organs. BOSTON – Patients with tuberous sclerosis complex, a genetic disorder characterized by the growth of noncancerous tumors in multiple organs of the body, have limited treatment options. A team led by investigators at Massachusetts General Hospital (MGH) has now […].
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