Aav gene therapies - Articles - Page 2

Samsung Life Science Fund invests in US ADC biotech Brickbio

March 19, 2024

Samsung Life Science Fund, created jointly by Samsung Biologics Co. Ltd., Samsung Biopeis Co. Ltd. and Samsung C&T Corp., said it invested in Waltham, Mass.-based antibody-drug conjugate (ADC) developer Brickbio Inc. on March 18, as its fifth biotech investment.

Concept art for adeno-associated viral-based gene therapy.

Drug Design, Drug Delivery & Technologies

New regulatory playbook to guide AAV gene therapy development for rare diseases

Feb. 7, 2024

The Foundation for the National Institutes of Health (FNIH) has announced the online publication of the first playbook designed to help accelerate the development of adeno-associated virus (AAV) gene therapies for rare diseases.

Cancer

Neuexcell's NXL-004 gets US orphan status for malignant glioma

Dec. 18, 2023

Neuexcell Therapeutics Inc. has announced that NXL-004, an investigational AAV gene therapy product being developed for the treatment of malignant glioma, has been awarded orphan drug designation by the FDA.

Inflammatory

Neutralizing hIL-33 shows efficacy in model of acute lung inflammation

July 14, 2023

Researchers from Astrazeneca plc have presented a novel adeno-associated virus (AAV)-delivered human (h)IL-33 neutralizing scFv-based protein construct at the 2023 World Congress on Basic and Clinical Pharmacology.

Ocular

Launch of Beacon Therapeutics with focus on gene therapies for retinal diseases

June 12, 2023

Beacon Therapeutics Holdings Ltd. has launched with a focus on developing a new generation of...

Endocrine/Metabolic

Preliminary results for NEU1-expressing gene therapy in preclinical sialidosis

May 29, 2023

Sialidosis is a lysosomal storage disease caused by mutations in the NEU1 gene, which encodes sialidase neuraminidase 1. These mutations lead to enzyme deficiency and subsequently accumulation of oligosaccharides and sialylated glycopeptides in tissues and body fluids, which in turn lead to cell and organ dysfunction. There are no approved therapies.
Three different AAV9 vectors encoding NEU1 were developed and tested by UMass Chan Medical School researchers in the preclinical setting in mice.

Endocrine/Metabolic

AAV9-CLN5 improves symptoms in mice with Batten disease

May 26, 2023

Neuronal ceroid lipofuscinosis, commonly known as Batten disease, is an inherited pediatric neurodegenerative lysosomal storage disease caused by mutations in the CLN5 gene. The disease is incurable and there is an urgent medical need for novel therapies. A murine model of Batten disease was developed to study a novel AAV vector that expresses CLN5, AAV9-CLN5. In the study by University College London investigators, the gene therapy, driven by the synapsin promoter, was intracerebroventricularly administered into neonatal Cln5-knockout mice.

Neurology/Psychiatric

Next-generation gene therapy shows superior efficacy for spinal muscular atrophy

May 25, 2023

Spinal muscular atrophy (SMA) is caused by mutations in the SMN1 gene, which encodes survival motor neuron 1, leading to reduced protein expression levels and degeneration of motor neurons in the spinal cord, with the consequent muscle atrophy. There is thus a need for new AAV gene therapies for SMA that confer better safety and efficacy.

Endocrine/Metabolic

AAV9-GLA restores α-galactosidase levels in murine model of Fabry disease

May 25, 2023

Fabry disease is a metabolic disease characterized by a deficiency in the lysosomal α-galactosidase enzyme caused by mutations in the GLA gene. This leads to substrate accumulation in the lysosomes, cellular dysfunction and organ damage.

Ear, Nose & Throat

SENS-501 demonstrates preliminary safety and efficacy in preclinical models

Feb. 16, 2023

Otoferlin is a calcium sensor protein critical for the transmission of the signal from inner hair cells (IHCs) to the spiral ganglion neurons (SGNs), and it is encoded by the OTOF gene. Pathogenic biallelic loss of function variations in OTOF result in failure of synaptic transmission, causing autosomal recessive deafness 9 (DFNB9), which is a congenital severe-to-profound auditory neuropathy.