Gene Therapy News

3 09, 2025

Avance Biosciences NGS Center of Excellence: Comprehensive Sequencing Solutions for Biologics, Gene, and Cell Therapy Development

2025-10-06T21:52:08+00:00

Avance Biosciences’ NGS Center of Excellence delivers comprehensive sequencing solutions, including short-read, long-read, and advanced single-cell platforms. Our regulatory-compliant workflows support biologics, gene, and cell therapy programs with high-quality data, enabling informed development...

Avance Biosciences NGS Center of Excellence: Comprehensive Sequencing Solutions for Biologics, Gene, and Cell Therapy Development2025-10-06T21:52:08+00:00
20 05, 2025

Groundbreaking Personalized Gene Therapy Saves Infant with Rare, Incurable Disease

2025-10-06T21:50:07+00:00

An NIH-supported team has delivered the first personalized CRISPR-based gene therapy to an infant with CPS1 deficiency, offering new hope for rare disease treatment. The customized approach shows early success and may pave the way for future individualized therapies...

Groundbreaking Personalized Gene Therapy Saves Infant with Rare, Incurable Disease2025-10-06T21:50:07+00:00
29 04, 2025

MIT Engineers Develop Gene Circuit for Precise Control of Gene Therapy

2025-10-06T21:50:08+00:00

MIT engineers have developed a gene circuit that precisely controls gene expression, offering potential breakthroughs in gene therapy for disorders like fragile X syndrome. This advancement helps ensure therapeutic genes are expressed at optimal levels, minimizing risks and paving the way for more effective treatments...

MIT Engineers Develop Gene Circuit for Precise Control of Gene Therapy2025-10-06T21:50:08+00:00
27 04, 2025

May 13-17 – American Society of Gene & Cell Therapy (ASGCT) Annual Meeting – 2025

2025-10-06T21:50:17+00:00

Avance Biosciences is excited to exhibit at the ASGCT 2025 conference in New Orleans, LA, from May 13–17, 2025. Join us to explore our expert bioanalytical solutions for gene and cell therapy development. Visit booth #750 to learn how we can support your therapeutic advancements...

May 13-17 – American Society of Gene & Cell Therapy (ASGCT) Annual Meeting – 20252025-10-06T21:50:17+00:00
26 02, 2025

Gene Therapy Restores Vision in Children with AIPL1-Related Retinal Dystrophy

2025-10-06T21:50:08+00:00

A groundbreaking gene therapy using rAAV8.hRKp.AIPL1 has restored vision in 11 children with AIPL1-related retinal dystrophy (LCA4), who were blind at birth. Published in The Lancet, the study shows significant improvements in visual acuity and retinal preservation, supporting expedited approval...

Gene Therapy Restores Vision in Children with AIPL1-Related Retinal Dystrophy2025-10-06T21:50:08+00:00
20 08, 2024

World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England

2025-10-06T21:52:07+00:00

Patients in England with severe beta-thalassaemia will be amongst the first in Europe to benefit from one-time gene therapy exagamglogene autotemcel. Exa-cel is the world’s first CRISPR-based gene therapy and the first gene therapy available in Europe for treating severe beta-thalassaemia...

World’s first gene editing therapy for blood disorder to be available to hundreds of patients in England2025-10-06T21:52:07+00:00
6 08, 2024

FDA Approves First T Cell Receptor (TCR) Gene Therapy

2025-10-06T21:50:09+00:00

Tecelra is the first FDA-approved T cell receptor (TCR) gene therapy. The product is an autologous T cell immunotherapy composed of a patient’s own T cells. T cells in Tecelra are modified to express a TCR that targets MAGE-A4, an antigen expressed by cancer cells in synovial sarcoma...

FDA Approves First T Cell Receptor (TCR) Gene Therapy2025-10-06T21:50:09+00:00
7 05, 2024

American Society of Gene and Cell Therapy (ASGCT) Conference – 2024

2025-10-06T21:50:18+00:00

Avance Biosciences™ is excited to announce our participation in the American Society of Gene & Cell Therapy (ASGCT) conference, where we’ll be showcasing our advanced services and expertise to propel the future of gene and cell therapy. [...]

American Society of Gene and Cell Therapy (ASGCT) Conference – 20242025-10-06T21:50:18+00:00
6 02, 2024

FDA issues final guidelines on incorporating human genome editing in gene therapy products

2025-10-06T21:52:08+00:00

The guidance provides recommendations regarding information that should be provided in an investigational new drug (IND) application to assess the safety and quality of the investigational GE product, including information on product design, product manufacturing and testing, nonclinical safety...

FDA issues final guidelines on incorporating human genome editing in gene therapy products2025-10-06T21:52:08+00:00
16 01, 2024

FDA’s Innovative Approach to Global Gene Therapy Collaboration

2025-10-06T21:50:09+00:00

In a landmark move, the FDA has initiated the Collaboration on Gene Therapies Global Pilot (CoGenT), signaling a significant leap toward worldwide collaboration in the field of gene therapy. This pilot aims to enable concurrent collaborative reviews of new gene therapy applications with global regulators…

FDA’s Innovative Approach to Global Gene Therapy Collaboration2025-10-06T21:50:09+00:00
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