Gene therapy Archives

News and Trends 12 Sep 2022

Sensorion receives positive opinion from EMA for treatment of hearing loss

The European Medicines Agency (EMA) through its Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion for a treatment for otoferlin gene mediated hearing loss. Clinical-stage biotech, Sensorion, announced the news today (September 12) that its application for an Orphan Drug Designation (ODD) for its lead therapy gene candidate, OTOF-GT will now be […]

September 12, 2022 - 3 minutesmins - By Liza Laws

Opinion 6 Sep 2022

Meeting tech transfer challenges when manufacturing new biopharmaceuticals

With increasingly complex therapeutics arriving almost monthly, efficient technology transfer between biopharma and manufacturers is more important than ever. Ken Forman, senior director of product strategy at IDBS, explains how good digital strategies can avoid common pitfalls in the tech transfer process. Biopharmaceutical lifecycle management (BPLM) is the key to delivering novel therapies and lifesaving […]

September 6, 2022 - 7 minutesmins - By External Contributor

Interview 31 Aug 2022

How CDMOs keep pace with trends in advanced therapies

Advanced therapies including cell and gene therapies are exploding in demand, but the manufacturing process can be difficult. Jean-Christophe Hyvert, president of biologics and cell & gene at the CDMO Lonza, sheds light on the latest trends in advanced therapies and how manufacturers are keeping up. In 2021, global funding going to cell and gene […]

August 31, 2022 - 8 minutesmins - By Jonathan Smith

News and Trends 31 Aug 2022

Research shows TikoMed’s ILB has the potential to treat neurodegenerative diseases including ALS

Peer-review research published in Frontiers in Pharmacology supports the broad spectrum mechanism of action of Swedish biopharma company TikoMed’s ILB neuroprotective drug platform. In multiple preclinical and clinical studies across a variety of neuroinflammation-driven diseases, the low molecular weight dextran sulfate compound both mobilized and modulated naturally occurring tissue repair mechanisms and restored cellular homeostasis […]

August 31, 2022 - 2 minutesmins - By Jim Cornall

News and Trends 29 Aug 2022

Strategic collaboration announced in China to accelerate the development of bacterial drugs

A strategic cooperation between Porton Advanced Solutions and Suzhou Royaltech Med Co., Ltd was announced today (August 29). The companies say the collaboration which was agreed earlier in the month (August 19) is set to integrate the resources and capabilities of both parties. The say it will jointly promote the establishment of microbial vectors used […]

August 29, 2022 - 2 minutesmins - By Liza Laws

News and Trends 26 Aug 2022

First gene therapy for adults with severe hemophilia approved by EC

The European Commission (EC) has granted conditional marketing authorization for a gene therapy to treat severe hemophilia A in adults it was announced yesterday (August 25). BioMarin Pharmaceutical Inc.’s Roctavian (valoctocogene roxaparvovec) treats the congenital factor 8 deficiency in adults patients without a history of factor VIII inhibitors without detectable antibodies to adeno-associated virus serotype […]

August 26, 2022 - 3 minutesmins - By Liza Laws

huntingtons disease dementia prilenia therapeutics

News and Trends 24 Aug 2022

Clearance given to clinical trial with potential to change course of brain disease

A trial to look at gene therapy, BV-01, for Huntington’s Disease has been given the go ahead to take place in France and has ‘the potential to change the course of the devastating disease. Asklepios BioPharmaceutical, Inc. (AskBio), a wholly owned and independently operated subsidiary of Bayer AG, received clearance for the phase 1/2 […]

August 24, 2022 - 3 minutesmins - By Liza Laws

beta-thalassemia blood APS antiphospholipid syndrome

News and Trends 18 Aug 2022

Patients with beta-thalassemia able to access first approved gene therapy

A gene therapy drug approved by the U.S. Food and Drug Administration (FDA) has been made accessible for people with beta-thalassemia. Zynteglo, a betibeglogene autoemcel, also known as beti-cel, treats adults and pediatric patients with the disease who need regular red blood (RBC) transfusions. The condition is passed down from one or both parents through […]

August 18, 2022 - 3 minutesmins - By Liza Laws

News and Trends 17 Aug 2022

AUM LifeTech secures $400,000 grant for lung cancer immunotherapy program

AUM LifeTech, Inc., a Philadelphia-based preclinical stage biotech company, has received a Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) of the National Institutes of Health (NIH) to advance its preclinical program in lung cancer immunotherapy using a non-viral RNA-targeting gene therapy approach. “This grant will help advance our preclinical immunotherapy […]

August 17, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 17 Aug 2022

New partnership delivers clinically proven products and manufacturing processes for advanced therapies

AGC Biologics has announced its partnership with supplier of human mesenchymal stem/stromal cells (hMSCs), RoosterBio Inc. The partnership, the companies say creates an end-to-end solution for the development and production of hMSCs and exosome therapeutics using RoosterBio’s well-established cell and media products and process development services together with AGC’s cell and gene therapy manufacturing capabilities. […]

August 17, 2022 - 3 minutesmins - By Liza Laws

News and Trends 16 Aug 2022

Remedium raises more funds for osteoarthritis gene therapy candidate

Remedium Bio, Inc., a biotech company developing novel gene therapies, has closed more than $2.3 million in its expanded seed round financing. Funding from the raise is being used to study Remedium’s lead product, a single-injection gene therapy potentially capable of reversing cartilage loss. The study is being undertaken in a collaboration with Tufts University […]

August 16, 2022 - 3 minutesmins - By Jim Cornall

News and Trends 8 Aug 2022

Allogenic immune cell therapies to be developed as three groups join forces

Three entities have joined forces to develop allogenic immune cell therapies based on a platform that generates functionally mature immune cells from induced pluripotent stem cells (iPSCs). ElevateBio LLC is a technology-driven company focused on powering transformative cell and gene therapies. It announced recently (August 5) that it has formed a yet-to-be-named company co-founded by […]

August 8, 2022 - 3 minutesmins - By Liza Laws

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Gene therapy

Sensorion receives positive opinion from EMA for treatment of hearing loss

Meeting tech transfer challenges when manufacturing new biopharmaceuticals

How CDMOs keep pace with trends in advanced therapies

Research shows TikoMed’s ILB has the potential to treat neurodegenerative diseases including ALS

Strategic collaboration announced in China to accelerate the development of bacterial drugs

First gene therapy for adults with severe hemophilia approved by EC

Clearance given to clinical trial with potential to change course of brain disease

Patients with beta-thalassemia able to access first approved gene therapy

AUM LifeTech secures $400,000 grant for lung cancer immunotherapy program

New partnership delivers clinically proven products and manufacturing processes for advanced therapies

Remedium raises more funds for osteoarthritis gene therapy candidate

Allogenic immune cell therapies to be developed as three groups join forces

Subscribe to our newsletter to get the latest biotech news!

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Illuminating biotech in the City of Light: Inside Paris’s thriving ecosystem

ASCO 2025: key highlights in cancer therapeutics

New report: Artificial intelligence and the future of antibody discovery

Stem cell therapy: a potential cure for hearing loss?

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

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