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
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
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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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
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
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
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
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.
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
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
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
Bio Pharma Dive
NOVEMBER 5, 2020
The setback for Bluebird's LentiGlobin comes amid a string of manufacturing-related delays for gene therapy developers
Bio Pharma Dive
OCTOBER 1, 2020
Gene therapy is no longer an approach for the future. It's a technique used now
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
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
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.
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.
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
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
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
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
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
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.
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
BioTech 365
DECEMBER 17, 2020
Scienmag
NOVEMBER 25, 2020
Medicine & Health Gene Therapy Genes Genetics Medicine/Health Ophthalmology
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.
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
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
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
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
BioSpace
JANUARY 25, 2021
Gene therapies offer great reward in the form of treating various devastating diseases, but there are also significant risks
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
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
Bio Pharma Dive
SEPTEMBER 10, 2020
The contract manufacturer is riding a boom in demand for both its gene therapy and vaccine manufacturing services
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
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
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
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.
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
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.
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
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
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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