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

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

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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

J&J pushes further into eye gene therapy

Bio Pharma Dive

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Scientists develop new gene therapy for eye disease

Scienmag

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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 […].