Author Archives: Marta Figueiredo PhD

CRISPR Partners with Capsida to Develop Gene-editing Therapies

CRISPR Therapeutics has joined forces with Capsida Biotherapeutics to discover and develop new gene-editing therapies for Friedreich’s ataxia (FA) and familial amyotrophic lateral sclerosis (ALS). The partnership seeks to combine CRISPR Therapeutics’ gene-editing expertise using its proprietary CRISPR/Cas9 platform with Capsida’s tissue-targeted, adeno-associated virus (AAV)-based gene therapies.

Center of Excellence Awarded Nearly $1.3M to Advance Research

The Friedreich’s Ataxia Center of Excellence of the Children’s Hospital of Philadelphia (CHOP) has been awarded a total of $1.275 million to advance research and clinical care of people with Friedreich’s ataxia (FA). The grant came from the Friedreich’s Ataxia Research Alliance (FARA), the Hamilton and Finneran families, and…

First In-human Trial of XCUR-FXN Planned for 2022

Exicure recently discussed preclinical research data and the next steps in developing XCUR-FXN, its investigational antisense oligonucleotide (ASO) therapy for Friedreich’s ataxia (FA), in a live webcast. After initiating investigational new drug (IND)-enabling studies of XCUR-FXN in late 2020 — intended to support regulatory clearance of clinical…

Exicure’s Program for Friedreich’s Ataxia Not Altered by COVID-19

The COVID-19 pandemic has not affected the preclinical program of XCUR-FXN, an experimental treatment for Friedreich’s ataxia (FA), Illinois-based Exicure said. Since biotechnology companies were designated essential businesses by the state’s governor J.B. Pritzker, Exicure will continue its research programs despite the the “stay-at-home” order in…

Leriglitazone Earns European Commission’s Orphan Drug Designation for FA

Minoryx Therapeutics’ lead investigational therapy leriglitazone (MIN-102) has been granted orphan drug designation by the European Commission for treating patients with Friedreich’s ataxia (FA). The European Commission’s decision follows a similar designation granted by the U.S. Food and Drug Administration in October. Orphan designation is given to investigative therapies…

Vibration Device Fails to Halt Motor Deterioration in Children with Friedreich’s Ataxia, Small Study Shows

Muscle stimulation through focal vibrations produced by a specialized device may prevent further deterioration of motor function in children with cerebellar ataxia, but not in Friedreich’s ataxia patients, according to a small study. The study, “Non-invasive Focal Mechanical Vibrations Delivered by Wearable Devices: An Open-Label Pilot Study…