Expanding Frontotemporal Dementia Gene Therapy to Both GRN and C9orf72 Mutations
William Chou, MD, president and chief executive officer of Passage Bio, discussed feedback from a recent Type C meeting with the FDA.
Evaluating Autologous Cord Blood Derived Cells for the Treatment of Preterm Infants
Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, discussed his lab’s research in the field of neonatal cell therapy.
Standardized Guidelines for Preclinical Testing and Cell Characterization are Critical to Cell Therapy Development
Atul Malhotra, MD, PhD, the head of the early neurodevelopment clinic at Monash Children's Hospital, discussed the importance of preclinical guidelines in the context of neonatal cell therapy.
New Findings May Help Bolster CRISPR/Cas9 Accuracy
David Taylor, PhD, an assistant professor in the department of molecular biosciences at UT Austin, discussed the Cas9 research conducted between 2 labs at UT Austin.
EU CHMP Gives Positive Opinion for Ide-Cel's Use in Multiple Myeloma
The recommendation comes after positive data was released from the recent phase 2 KarMMa trial.
Gene Editing Trial Underway in Pediatric Methylmalonic Acidemia
The therapy utilizes LogicBio’s GeneRide technology to insert a corrective copy of the target gene via synthetic AAV vectors.
