Gene therapy Archives

Best in Biotech 30 Aug 2023

Five North Carolina biotech companies making their mark

North Carolina is home to Research Triangle Park, an R&D sanctuary situated between the cities of Raleigh and Durham, which just happens to be one of the biggest biotech hubs in the U.S., with a multitude of renowned companies. The southeastern state even rivals the likes of Boston, San Francisco, Los Angeles, and San Diego, […]

August 30, 2023 - 5 minutesmins - By Willow Shah-Neville

In Depth 27 Jul 2023

Cell and gene therapy manufacturing: trends, advancements and challenges

Cell and gene therapy manufacturing has been a pertinent topic for a while now, largely due to the fact it is such a complicated process, involving a number of moving parts and excessive costs. As such, figuring out how to make these types of therapies scalable, less time consuming and cost-effective is still an ongoing […]

July 27, 2023 - 7 minutesmins - By Willow Shah-Neville

In Depth 19 Jul 2023

Genetic engineering giants: is China poised to lead the way?

For many people, when they hear China and genetic engineering in the same sentence, it is often synonymous with scandal, and gene-edited babies may spring to mind. And, although it is true that nearly five years ago, researcher He Jiankui infamously claimed he had created the first ever gene-edited babies, before going to prison for […]

July 19, 2023 - 7 minutesmins - By Willow Shah-Neville

News and Trends 13 Jul 2023

Gene therapy company EG 427 raises €18M

EG 427, a French biotechnology company leading the development of pinpoint DNA medicine solutions, has announced the final closing of a Series A financing. The gene therapy company raised an additional €5 million ($5.6 million) in the final closing from a combination of existing investors and new family office investors, bringing the total raised in […]

July 13, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 11 Jul 2023

NK:IO awarded £1.6M grant to develop NK cell therapy

NK:IO, a U.K. company working on natural killer (NK) cell biology applied to the development of exceptionally potent, off-the-shelf cell therapies targeting solid tumors, has been awarded £1.6 million ($2.1 million) in grant funding from Innovate UK’s New Cancer Therapeutics program. The funding will support preclinical development, including development of a manufacturing process in collaboration […]

July 11, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 30 Jun 2023

FDA approves BioMarin gene therapy for hemophilia A

BioMarin Pharmaceutical Inc. has announced that the United States Food and Drug Administration (FDA) has approved ROCTAVIAN (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test. The […]

June 30, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 27 Jun 2023

Astraveus exits stealth to advance cell and gene therapy platform

Astraveus SAS has emerged from stealth with the completion of an oversubscribed €16.5 million ($18.1 million) Series Seed financing. The company creates modular, microfluidic cell foundries that transform cell and gene therapy (CGT) manufacturing. Astraveus has developed the Lakhesys platform, an end-to-end cell foundry that uses deep process optimization and single-use, microfluidic bioprocessors to deliver […]

June 27, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 23 Jun 2023

Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Sarepta Therapeutics, Inc. has announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene moxeparvovec-rokl), an Adeno-associated virus (AAV)-based gene therapy for the treatment of ambulatory pediatric patients aged 4 through 5 years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene. This indication is approved under accelerated approval […]

June 23, 2023 - 3 minutesmins - By Jim Cornall

In Depth 19 Jun 2023

Gene therapy: a promising solution for sickle cell disease

Sickle cell disease is debilitating, and many people who suffer with it find that their lives are defined by pain. This means a curative treatment would be completely life–altering, and, as we observe World Sickle Cell Day on June 19, we explore how gene therapy could be the most promising solution for curing sickle cell […]

June 19, 2023 - 6 minutesmins - By Willow Shah-Neville

Beyond Biotech podcast gene cell therapy

Podcast 16 Jun 2023

Beyond Biotech podcast 50

The International Society for Cell & Gene Therapy (ISCT), the global society of more than 3,000 members dedicated to the translation of cell and gene therapies (CGT) into safe and effective treatments to improve patient lives, recently hosted the largest translation-focused event connecting academic, regulatory and industry experts from around the world to collaborate and […]

June 16, 2023

News and Trends 8 Jun 2023

Kate Therapeutics launches with $51M to develop genetic medicines for muscle and heart diseases

Gene therapy company Kate Therapeutics Inc. (KateTx), has emerged from stealth mode with a $51 million Series A financing. In addition, the company has granted Astellas Pharma Inc. an exclusive, worldwide license to develop, manufacture and commercialize KT430 to treat X-linked myotubular myopathy (XLMTM). There are a large number of genetically defined and complex muscle […]

June 8, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 7 Jun 2023

AAVantgarde closes €61M financing to move gene delivery program to the clinic

AAVantgarde Bio, a clinical-stage, Italian-based international biotechnology company with two proprietary adeno-associated viral (AAV) vector platforms for large gene delivery, has closed a €61 million ($65.1 million) Series A financing. The amount represents the largest series A round ever in Italian biotech. AAVantgarde has two proprietary, AAV-based large gene delivery platforms: one leveraging DNA recombination, […]

June 7, 2023 - 3 minutesmins - By Jim Cornall

Knowledge hub

Categories

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Approaches

Therapeutic areas

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

R&D

BD

World biotech leaders

Connect with decision-makers and researchers worldwide for collaborative opportunities

Company

Join Us

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Gene therapy

Five North Carolina biotech companies making their mark

Cell and gene therapy manufacturing: trends, advancements and challenges

Genetic engineering giants: is China poised to lead the way?

Gene therapy company EG 427 raises €18M

NK:IO awarded £1.6M grant to develop NK cell therapy

FDA approves BioMarin gene therapy for hemophilia A

Astraveus exits stealth to advance cell and gene therapy platform

Sarepta gets approval for first gene therapy for Duchenne muscular dystrophy

Gene therapy: a promising solution for sickle cell disease

Beyond Biotech podcast 50

Kate Therapeutics launches with $51M to develop genetic medicines for muscle and heart diseases

AAVantgarde closes €61M financing to move gene delivery program to the clinic

Subscribe to our newsletter to get the latest biotech news!

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

The top biotech deals of March 2025

The biggest biotech funding rounds in March 2025

Join the webinar: AI in antibody discovery - from in silico analysis to AI-enabled data management

Beyond amyloid: The search for the next big thing in Alzheimer’s treatment

Connect with decision-makers and researchers worldwide for collaborative opportunities

Subscribe to our newsletter

Advertise with us

Subscribe to our newsletter

Get the latest biotech news directly to your inbox!

Newsletter Signup - Footer

Subscribe to our newsletter to get the latest biotech news!