Lysosomal Disorders

With advancements happening at unprecedented rates, these are the cell and gene therapy companies and pipelines we’re keeping a close eye on.

LogicBio's Mariana Nacht, PhD, shares details of the company's novel capsid discovery platform and gene editing technology.

Cynata Therapeutics' Ross Macdonald, PhD, discusses the company's pipeline and clinical-phase trials.

Ross Macdonald, PhD, managing director and CEO of Cynata Therapeutics, discusses the biotech company's approach to overcoming reproducibility challenges with mesenchymal stem cells.

Maria A. Croyle, PhD, discusses the development of a novel film matrix that can safely and effectively store and transport AAV-based gene therapies.

Maria A. Croyle, PhD, discusses the manufacturing challenges facing the gene therapy space and her inspiration for developing a novel preservation method for viral vector-based therapies.

Typically treated with stem cell transplantation, this lentiviral vector-based gene therapy approach may resolve challenges related to donor availability and post-transplantation complications.

An autologous lentiviral-based gene therapy demonstrated significant benefit, including high overall and event-free survival rates, immune reconstitution, and metabolic correction, for patients with ADA-SCID.

The full regulatory approval of agalsidase beta has blocked the accelerated approval pathway for AVR-RD-01 as a treatment for patients with Fabry disease.

The first patient has been enrolled and treated with RGX-121 in the third cohort of a phase 1/2 study for patients up to 5 years old with severe MPS II.

Orchard Therapeutics released its plans for 2021, which included the launch of OTL-200 (Libmeldy) in Europe and development of an FDA submission strategy in the United States.

Descriptive findings from the phase 1/2 study allude to promise for the gene therapy in the rare pediatric disorder.

New phase 2/3 findings meet investigators' hypotheses on the gene therapy's mixed benefits for heparan sulfate reduction.

The AAV-based gene therapy was well-tolerated and showed enough promising effect to warrant further investigation, researchers reported.

Investigators observed sustained, dose-dependent benefits for young patients at 24 months.

The investigators found the primary efficacy endpoints occurred in 20 evaluable patients (87%).

FLT201 is a combination of AAVS3 capsid and a liver-specific promotor to drive the expression of GCasevar85.

New WORLDSymposium findings show a treated patient had 100% reduction in the accumulating fatty substrate associated with the burdensome rare disease after 1 year.

These data follow a recent announcement from Sio Gene Therapies that the first patient in the high-dose cohort has been dosed.

Interim data from the first 8 pediatric patients showed that the AAV-CLN6 gene therapy demonstrated a positive impact on motor and language function compared to a natural history dataset, as well as in comparison to in-study sibling pairs.

Abeona Therapeutics is currently planning a phase 1/2 clinical trial to evaluate ABO-202 in Batten disease.

New research highlights the potential of fetal gene therapy to prevent and cure neonatal lethal neurodegenerative diseases in humans in utero.

The phase 1/2 trial for ABO-102 (AAV-SGSH), clinical gene therapy for the treatment of Sanfilippo syndrome type A (MPS III A) shows efficacy in trial update.

Orchard Therapeutics announced that its gene therapy candidate, OTL-200, has been granted Rare Pediatric Disease designation for the treatment of metachromatic leukodystrophy.

REGENXBIO Inc. announced that the U.S. FDA has granted Fast Track designation to RGX-131, a novel, one-time investigational treatment for mucopolysaccharidosis type II (MPS II).

The U.S. FDA has cleared the Investigational New Drug application for DTX401 for the treatment of glycogen storage disease type Ia (GSDIa).

Abeona announced that the U.S. FDA has granted rare pediatric disease designation to ABO-202, a gene therapy in development for the treatment of infantile and late infantile-onset Batten disease.

Abeona Therapeutics, Inc. announced that the FDA has granted Orphan Drug Designation to its ABO-202 program for the treatment of infantile Batten disease.

Biotechnology company Avrobio has completed a $60 million Series B financing to advance multiple gene therapies, including AVR-RD-01, a proposed single-dose lentiviral gene therapy for Fabry disease (FD).