Gene therapy Archives

News and Trends 8 May 2023

How thalassemia gene therapy approval is making a difference

A newly-approved gene therapy is transforming the lives of patients with beta thalassemia – an inherited blood disorder – by helping to stop or significantly reduce their reliance on blood transfusions, says an expert from a leading global health system, Cleveland Clinic, on International Thalassemia Day on May 8. A gene therapy known as betibeglogene […]

May 8, 2023 - 5 minutesmins - By Jim Cornall

News and Trends 25 Apr 2023

Gene therapy and chemotherapy could be new osteosarcoma treatment option

New research has identified a potential therapeutic target and developed a unique delivery system to treat osteosarcoma, a bone cancer that primarily affects children and adolescents. The standard-of-care treatment plan for osteosarcoma today is no different to when first introduced almost 50 years ago. However, nearly one third of patients’ relapse and need new interventions. […]

April 25, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 24 Apr 2023

Life Biosciences gene therapy data shows visual function restored in primates

Life Biosciences (Life Bio) has announced preclinical data in nonhuman primates (NHP) for its novel gene therapy candidate, which uses a partial epigenetic reprogramming approach to restore visual function. The approach has been shown to reverse aging, improve vision, and extend lifespan in mice, but whether epigenetic reprogramming would work in primates was not known. […]

April 24, 2023 - 4 minutesmins - By Jim Cornall

In Depth 18 Apr 2023

Gene therapy: a powerful tool for hemophilia B?

The approval of HEMGENIX, the first-ever gene therapy for hemophilia B, by the U.S. Food and Drug Administration (FDA) followed by the European Commission, has been momentous in rare disease therapeutic research, paving the way for various gene therapies that are currently being studied. As we observed World Hemophilia Day on April 17, let us […]

April 18, 2023 - 10 minutesmins - By Roohi Mariam Peter

News and Trends 18 Apr 2023

Complement Therapeutics raises €72M for eye condition gene therapy

Complement Therapeutics GmbH (CTx), a preclinical stage biotechnology company developing novel therapeutics for complement mediated diseases, has completed a €72 million ($78.7 million) Series A financing. The round was led by Gimv, a Belgian-based private equity and venture capital fund, co-led by Forbion as existing investor and further joined by BioGeneration Ventures (BGV), Panakes Partners, […]

April 18, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 5 Apr 2023

Ginkgo Bioworks boosts gene therapy capacity with StrideBio asset acquisition

Ginkgo Bioworks has acquired StrideBio’s adeno-associated virus (AAV) capsid discovery and engineering platform assets. A secondary close is scheduled upon the transfer of certain additional in-license agreements to Ginkgo Bioworks. Ginkgo Bioworks will incorporate the assets and IP into its end-to-end AAV gene therapy development platform, allowing the company’s customers to leverage new tools to […]

April 5, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 4 Apr 2023

New research may lead to gene therapies for kidney disease

Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease—about a half million people in the U.S. alone suffer from the condition. There is currently no cure, but new research could open the door to new gene therapies for treating most cases of this kidney disease. For several decades, researchers have […]

April 4, 2023 - 5 minutesmins - By Jim Cornall

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

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Gene therapy

How thalassemia gene therapy approval is making a difference

Gene therapy and chemotherapy could be new osteosarcoma treatment option

Life Biosciences gene therapy data shows visual function restored in primates

Gene therapy: a powerful tool for hemophilia B?

Complement Therapeutics raises €72M for eye condition gene therapy

Ginkgo Bioworks boosts gene therapy capacity with StrideBio asset acquisition

New research may lead to gene therapies for kidney disease

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

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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