Gene therapy Archives

News and Trends 30 Mar 2023

Could new riboswitch make gene therapy safer?

Turning genes on and off as easily and predictably as flicking a switch could be a powerful tool in medicine and biotech. A type of technology called a riboswitch might be the key.The Okinawa Institute of Science and Technology (OIST) in Japan, in collaboration with Astellas Pharma Inc., has developed a new toolkit that uses […]

March 30, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 16 Mar 2023

Inspire Biotherapeutics launches to target lung diseases through gene therapy

Inspire Biotherapeutics Inc., an emerging Canadian biotech company with a mission to create gene therapies for monogenic and acquired diseases of the lung, is set to move its novel lung tropic AAV gene therapy into clinical validation. The AAVenger platform has demonstrated transduction and long-term expression in lung tissue without impact on other tissue types […]

March 16, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 15 Mar 2023

Nanite receives funding to deliver genetic therapies to the lungs

Nanite Inc. has announced an investment from the Cystic Fibrosis Foundation of up to $2 million to develop gene delivery technologies for the lung. This builds on the $6 million seed financing recently announced by the company. One of the most significant challenges in developing new therapies for patients with cystic fibrosis is effective drug […]

March 15, 2023 - 2 minutesmins - By Jim Cornall

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

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

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Gene therapy

Could new riboswitch make gene therapy safer?

Inspire Biotherapeutics launches to target lung diseases through gene therapy

Nanite receives funding to deliver genetic therapies to the lungs

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

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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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