Krystal Biotech Inc. has received IND clearance from the FDA for KB-408 for the treatment of α1-antitrypsin deficiency (AATD). KB-408 is a modified, replication-defective, nonintegrating HSV-1-derived vector carrying two full-length copies of the serpin family A member 1 gene (SERPINA1) to enable expression of α1-antitrypsin (AAT).

Atamyo Therapeutics SAS has filed a clinical trial application (CTA) in Europe for ATA-200, its gene therapy targeting γ-sarcoglycan (SGCG)-related limb-girdle muscular dystrophy type 2C/R5 (LGMD-R5).

Alveogene Ltd. has launched with a focus on inhaled gene therapies for respiratory disorders. The company has been created by Oxford Science Enterprises, Harrington Discovery Institute at University Hospitals, and Old College Capital in partnership with six scientists from the UK Respiratory Gene Therapy Consortium (GTC).

Biomarin Pharmaceutical Inc. has reported progress across its research and development portfolio, including early-stage product candidates.

Bloomsbury Genetic Therapies Limited has disclosed a new pipeline program – BGT-PD...

Scientists behind Purespring Therapeutics Ltd. have made progress in overcoming the problems of accessibility, complexity of structure and diversity of cell types that have held back the use of gene therapy in the kidneys, reporting success in treating nephrotic syndrome in a mouse model of the inherited form of the renal disease.

Non-profit Solve GNE LLC has raised over $2.5 million and announced sponsored research agreements to help advance research in hereditary inclusion body myopathy (HIBM), or GNE myopathy (GNEM).

Using whole genome sequencing, scientists at Boston Children’s Hospital have studied the genes and mutations of ataxia-telangiectasia (A-T) that would respond to treatments with splice-switching antisense oligonucleotides (ASOs). Their work, published on July 12, 2023, in Nature, determined the appropriate individualized genetic therapy for these patients and identified a new drug.