All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
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Features in this in-depth focus highlight some of the important developments in the cell and gene therapy market, as well as the key manufacturing strategies to consider when taking them from concept to commercialisation.
A newly developed investigational microneedle patch caused mice to regrow hair faster than minoxidil, a leading alopecia treatment.
The Parkinson’s disease market is set to triple in sales by 2029 driven by novel biologics and delivery systems, reports GlobalData.
Ritlecitinib was shown to reduce scalp hair loss to less than or equal to 20 percent after six months in Pfizer’s Phase IIb/III trial.
AstraZeneca’s Saphnelo (anifrolumab-fnia) has been approved in the US for patients with moderate to severe systemic lupus erythematosus.
The Phase II study of Evrysdi showed significant improvements in survival and motor milestones in babies with type 1 spinal muscular atrophy (SMA).
Biogen and Ionis announced positive topline data on investigational Alzheimer’s treatment, indicating 50 percent tau protein reduction.
The Committee for Medicinal Products for Human Use (CHMP) recommended two medicines for approval and provided a negative opinion on Nouryant.
Eli Lilly and Company has announced the acquisition of California-based biotech company, Protomer Technologies.
The ISSCR has released updated guidelines for stem cell research and clinical translation of stem cell-based therapies.
JadiCell™ was shown to significantly improve survival in COVID-19 patients with Acute Respiratory Distress Syndrome (ARDS) and also reduce lung scarring associated with long-COVID-19.
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
Transplanting stem cells directly into the trachea of preterm infants with bronchopulmonary dysplasia was found to be safe and of some benefit in the most vulnerable group.
European Pharmaceutical Review’s Hannah Balfour explores the findings of the Cell and Gene Therapy (CGT) Catapult’s 2020 advanced therapy medicinal product trials report and discusses how the European and global CGT markets are expected to perform in coming years.