Abcellera Biologics Inc. has received a no objection letter from Health Canada authorizing its clinical trial application (CTA) for ABCL-635.

The FDA has granted orphan drug designation to Cure Rare Disease’s CRD-002, an antisense oligonucleotide therapeutic for the treatment of spinocerebellar ataxia (SCA), including spinocerebellar ataxia type 3 (SCA3).

Atyr Pharma Inc. has advanced ATYR-0101 into IND-enabling studies for pulmonary fibrosis, and is targeting an IND application in the second half of next year.

Metabolic disorders such as argininosuccinic and glutaric aciduria, methylmalonic acidemia, homocystinuria or primary hyperoxaluria require specific diets to prevent the accumulation of substances that the body can’t process. Current treatments mainly focus on managing symptoms and metabolite levels, and do not always prevent the progressive deterioration caused by mutations associated with the condition. However, emerging gene therapies hold promise for transforming these diseases by targeting their underlying causes, as presented in the oral abstract session, “Gene and cell therapy for metabolic diseases” of the ongoing 28th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT) meeting in New Orleans.

The U.S. FDA’s decision to phase out animal testing for INDs is driving a new market of alternative, nonanimal testing technologies like organoids and organs-on-a-chip, speakers at Bio Korea 2025 said.