The U.S. FDA has cleared Tessera Therapeutics Inc.’s IND application for TSRA-196, its lead in vivo gene editing therapy for α-1 antitrypsin deficiency (AATD). The Australian Human Research Ethics Committee (HREC) has also granted approval for the company to begin the phase I/II clinical study.
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
Using a customized gene editing therapy, researchers at the Children’s Hospital of Philadelphia have reported success in treating an infant with a severe metabolic disorder. Kiran Musunuru, Barry J. Gertz Professor for Translational Research in the University of Pennsylvania’s Perelman School of Medicine, presented the case at the American Society of Gene and Cell Therapy’s 2025 annual meeting. The case study was simultaneously published in The New England Journal of Medicine.
Yoltech Therapeutics Co. Ltd. has advanced YOLT-204 into the clinic for the treatment of transfusion-dependent β-thalassemia (TDT). If successful, YOLT-204 may provide an off-the-shelf curative treatment for TDT patients without conditioning chemotherapy and hematopoietic stem cell transplantation.
Arbor Biotechnologies Inc. has gained IND clearance from the FDA for ABO-101, a novel gene editing therapeutic designed to address primary hyperoxaluria type 1 (PH1). A phase I/II study in adult and pediatric patients with PH1 is expected to begin in the first half of 2025.
Researchers from Proqr Therapeutics NV recently presented preclinical data on AX-0810, a single-stranded RNA editing oligonucleotide (EON) targeting RNA coding for NTCP cotransporter
Precision Biosciences Inc. has submitted its first clinical trial applications (CTAs) to initiate a phase I study evaluating PBGENE-HBV, an in vivo gene editing program designed to potentially cure chronic hepatitis B virus (HBV).
Metagenomi Inc. has reported data from an ongoing preclinical study designed to provide evidence supporting the potential durability and safety of the company’s hemophilia A gene editing investigational therapy, MGX-001.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
New single-step genome editing techniques that enable the insertion, inversion or deletion of long DNA sequences at specified genome positions have been demonstrated in bacteria.
