Gene therapy Archives

Interview 8 Mar 2023

Phacilitate’s Women in Advanced Therapies program supports women in science

Marlin Frechette is the chief quality and compliance officer at FUJIFILM Irvine Scientific. But she has another role. Frechette is also a member of Phacilitate’s Women in Advanced Therapies program, and was formerly a mentor, and she tells us here how the program supports women in executive level life science roles. Frechette takes up the […]

March 8, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 3 Mar 2023

Beyond Biotech podcast 36: Bio-Europe Spring and Cellular Origins

Two of the interviews on the podcast today are related to Bio-Europe Spring. There are conversations with Rosie Bernard, senior director, production and content strategy for EBD Group EU, with an overview of the event, and also Jordan Stillman, project manager, partnering services at EBD Group. We also have a chat with Cellular Origins’ CEO […]

March 3, 2023

Interview 3 Mar 2023

Interview: how has the first gene therapy for hemophilia B transformed research?

Hemophilia B, a rare disease that affects more than 230,000 people worldwide is caused by the lack of or defective clotting protein factor IX often due to a spontaneous mutation in the F9 gene, leading to excessive bleeding. Until recently, routine prophylactic infusions of factor IX replacement therapy to maintain enough clotting factor to prevent […]

March 3, 2023 - 8 minutesmins - By Roohi Mariam Peter

News and Trends 3 Mar 2023

Porton and DanausGT look to boost development of gene and cell therapy

Porton Advanced Solutions Ltd. and DanausGT Biotechnology Co., Ltd. have announced a strategic collaboration in gene and cell therapy pipelines to expedite the development of innovative therapeutics. As an end-to-end gene and cell therapy service provider, Porton Advanced CDMO offers solutions covering plasmids, cell therapy, gene therapy, oncolytic viruses, mRNA therapy, and bacterial therapy. DanausGT […]

March 3, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 2 Mar 2023

New lipid nanoparticle CRISPR delivery system developed

Lipid nanoparticles have been used to encapsulate CRISPR-Cas9 and deliver it to cells in mice, where it was highly effective at knocking down expression of a target protein. Gene therapy is a potential mode of treatment for a wide variety of diseases caused by genetic mutations. While it has been an area of diverse and […]

March 2, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 28 Feb 2023

European Commission approves first gene therapy for hemophilia B

The European Commission has approved the first gene therapy for the treatment of hemophilia B developed by biotech company CSL Behring. The one-time gene therapy etranacogene dezaparvovec sold under the brand name Hemgenix is manufactured by UniQure Inc. and distributed by CSL Behring. The gene therapy is used to treat severe and moderately severe hemophilia […]

February 28, 2023 - 3 minutesmins - By Roohi Mariam Peter

Opinion 28 Feb 2023

Life changing treatments for rare disease – a beacon of hope

By Sander Slootweg, managing partner, Forbion More than 300 million people around the globe are affected by a rare disease. This number does not even take into account the impact on these individuals’ support units that includes family, friends and caregivers. A rare disease is defined as a disease that affects no more than 1 […]

February 28, 2023 - 6 minutesmins - By External Contributor

News and Trends 17 Feb 2023

Report: China leads the way with phase 1 studies

Novotech, an Asia Pacific biotech CRO, has published a new global report on phase 1 trial activity including current metrics on fast growth regions and therapeutic areas. The data analysis found that Asia Pacific has been the fastest-growing region for phase 1 clinical trials in the last 10 years, reaching a share of 58% in […]

February 17, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 Feb 2023

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Rocket Pharmaceuticals, Inc says the U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) designation to RP-A501. RP-A501 is Rocket Pharmaceuticals’ investigational adeno-associated virus (AAV)-based gene therapy for the treatment of Danon disease, a devastating and fatal genetic cardiac disease for which there are no disease-altering therapies available. RMAT designation was […]

February 10, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 9 Feb 2023

Cell and Gene Therapy Catapult boost for life science campus

The Cell and Gene Therapy Catapult (CGT Catapult), an independent U.K. innovation and technology organization specializing in the advancement of cell and gene therapies, has announced its involvement in the development of a major new campus for life science businesses in Stevenage. The new campus was announced by UBS Asset Management’s Real Estate & Private […]

February 9, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 31 Jan 2023

Sensorion and EVEON collaborating on injection system to deliver gene therapy inner ear treatments

Sensorion and EVEON are collaborating on the design and development of an injection system for the delivery of Sensorion’s gene therapy products to the inner ear. Therapies for treating genetic hearing loss are often challenging due to the need for delivery into the inner ear, a very small and delicate organ embedded in the temporal […]

January 31, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 24 Jan 2023

Funding for university to develop new motor neuron disease therapies

Medical research charity LifeArc, the Motor Neurone Disease Association (MND Association) and the My Name’5 Doddie Foundation have jointly awarded £1 million ($1.2 million) to researchers at University College London (UCL) to progress two pioneering new therapies that could help transform the lives of those living with motor neuron disease (MND). The research grants are […]

January 24, 2023 - 4 minutesmins - By Jim Cornall

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Gene therapy

Phacilitate’s Women in Advanced Therapies program supports women in science

Beyond Biotech podcast 36: Bio-Europe Spring and Cellular Origins

Interview: how has the first gene therapy for hemophilia B transformed research?

Porton and DanausGT look to boost development of gene and cell therapy

New lipid nanoparticle CRISPR delivery system developed

European Commission approves first gene therapy for hemophilia B

Life changing treatments for rare disease – a beacon of hope

Report: China leads the way with phase 1 studies

Rocket Pharmaceuticals receives FDA RMAT designation for Danon disease gene therapy treatment

Cell and Gene Therapy Catapult boost for life science campus

Sensorion and EVEON collaborating on injection system to deliver gene therapy inner ear treatments

Funding for university to develop new motor neuron disease therapies

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

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