Gene therapy Archives

News and Trends 1 Jun 2023

ElevateBio raises $401M for cell and gene therapies

ElevateBio, LLC (ElevateBio), a technology-driven company focused on cell and gene therapies, has closed its $401 million Series D financing. “We have made significant strides in scaling our technologies and end-to-end capabilities in our pursuit to become the world’s most indispensable cell and gene therapy technology company. We are thrilled to welcome Khalil to our […]

June 1, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 26 May 2023

Novartis buys AVROBIO cystinosis gene therapy program for $87.5M

AVROBIO, Inc., a clinical-stage gene therapy company working to free people from a lifetime of genetic disease, has agreed to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis for $87.5 million in cash. AVROBIO retains full rights to its portfolio of first-in-class HSC gene therapies for […]

May 26, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 26 May 2023

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

Rocket Pharmaceuticals, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RP-L301, the company’s investigational lentiviral-based gene therapy for pyruvate kinase deficiency (PKD). PKD is a rare blood disorder characterized by severe anemia and excessive red blood cell breakdown. RMAT designation was granted based […]

May 26, 2023 - 4 minutesmins - By Jim Cornall

News and Trends 22 May 2023

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

The U.S. Food and Drug Administration has approved Vyjuvek, a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy, for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene. The approval of Vyjuvek was granted to […]

May 22, 2023 - 3 minutesmins - By Jim Cornall

In Depth 19 May 2023

Can cardiovascular diseases be cured? The future of heart treatment

Cardiovascular diseases, characterized as a group of disorders of the heart and blood vessels, are the leading cause of death worldwide. In fact, in the U.S. alone, it is thought that one person dies every 33 seconds from cardiovascular disease. Although clearly a huge health problem throughout the world, developing cures for cardiovascular diseases is […]

May 19, 2023 - 11 minutesmins - By Willow Shah-Neville

News and Trends 18 May 2023

8 rare diseases selected for gene therapy trial portfolio

The Foundation for the National Institutes of Health (FNIH) says the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio. This portfolio pioneers a novel approach to gene therapy development for rare diseases by demonstrating that manufacturing and testing standards can provide a streamlined approval […]

May 18, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 18 May 2023

Sania Therapeutics launches to advance neural circuit dysfunction medicines

Sania Therapeutics, which develops genetic medicines for neural circuit dysfunction, has launched with the unveiling of its platforms at the American Society of Gene & Cell Therapy (ASGCT) conference. Sania Therapeutics’ approach combines the evolution of adeno-associated viruses (AAV) in human neural circuits, the fundamental components of the nervous system, and tuneable conditionally-activated ion channels. […]

May 18, 2023 - 2 minutesmins - By Jim Cornall

News and Trends 12 May 2023

Cell and gene therapy company NewBiologix exits stealth with $50M

NewBiologix SA has emerged from stealth as a technology innovation company developing a proprietary and breakthrough platform for the advanced engineering of cell lines used to manufacture gene and cell therapies. The company’s DNA-based platform is being developed to address key manufacturing challenges associated with the production of recombinant adeno-associated virus (rAAV) vectors used to […]

May 12, 2023 - 3 minutesmins - By Jim Cornall

News and Trends 10 May 2023

Delivering on the need for vector innovation in genetic medicine

By Tuyen Ong, CEO Ring Therapeutics, CEO-Partner Flagship Pioneering Gene therapy is a life-changing class of medicine that can treat patients with once intractable diseases. While revolutionary, significant limitations remain for the vectors that carry them. Viruses are masters at delivering genetic material to the nucleus of the organisms they infect, and because of this, […]

May 10, 2023 - 2 minutesmins - By External Contributor

News and Trends 10 May 2023

Cell rejuvenation for age-related diseases

By Sharon Rosenzweig-Lipson, CSO, Life Biosciences Cellular rejuvenation is quickly becoming a hot topic in the development of novel therapeutic modalities for a range of diseases. The leading approaches to cellular rejuvenation currently in development utilize epigenetic reprogramming to achieve this goal. Epigenetics is the study of how positive and negative lifestyle factors (exercise, smoking, […]

May 10, 2023 - 3 minutesmins - By External Contributor

Opinion 9 May 2023

Where is innovation needed in cell therapy?

By Dr Ryan Roberts, cell processing lead at Cellular Origins Despite the rapidly emerging innovations in the field of cell and gene therapy, bottlenecks in cell therapy manufacturing processes are preventing these transformative medicines from reaching patients. There are several broad challenges to the scalability and affordability of cell therapies, including a reliance on manual […]

May 9, 2023 - 5 minutesmins - By External Contributor

News and Trends 9 May 2023

Gene & cell therapy manufacturer Ascend launches with more than $130M

Ascend Gene & Cell Therapies (Ascend), a company innovating manufacturing processes to improve quality, potency, safety and cost enabling better reach for gene therapy technologies, has launched as a facilitator for gene therapy companies to help translate product ideas into clinical programs. Ascend Gene & Cell Therapies offers process development, clinical manufacturing, and analytical and […]

May 9, 2023 - 2 minutesmins - By Jim Cornall

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Gene therapy

ElevateBio raises $401M for cell and gene therapies

Novartis buys AVROBIO cystinosis gene therapy program for $87.5M

Rocket Pharmaceuticals receives FDA designation for pyruvate kinase deficiency gene therapy

FDA approves first topical gene therapy for dystrophic epidermolysis bullosa wound treatment

Can cardiovascular diseases be cured? The future of heart treatment

8 rare diseases selected for gene therapy trial portfolio

Sania Therapeutics launches to advance neural circuit dysfunction medicines

Cell and gene therapy company NewBiologix exits stealth with $50M

Delivering on the need for vector innovation in genetic medicine

Cell rejuvenation for age-related diseases

Where is innovation needed in cell therapy?

Gene & cell therapy manufacturer Ascend launches with more than $130M

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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