HEMOPHILIA

ABioMarin's gene therapy Roctavian has gained FDA approval for the treatment of severe hemophilia A in adults. Priced at $2.9 million, the therapy is suitable for around2,500 US patients. Clinical trials reported a 52% reduction in annual bleeding cases,though this efficacy data is lower than the EU approval's 85.5%. The single-dose therapy represents a convenient and promising addition to the treatment landscape,potentially expanding access to treatment for a broader range of patients with hemophilia A

SICKLE CELL DISEASE (SCD)

A recent clinical trial published in the New EnglandJournal of Medicine demonstrated a decrease in painfulvaso-occlusive events in SCD patients. The patients were treated with stem cell gene therapy, using CRISPR-Cas9 technology to edit genes and increasefetal hemoglobin production. These results demonstrate a promising curative treatment

NEURONAL CEROID LIPOFUSCINOSIS TYPE 2

Regenxbio presented Initial interim data from a single patient trial of its investigational gene therapy, RGX-181(AAV9 delivering TPP1) gene therapy at SSIEM 2023. The therapy aims to treat late-infantile neuronal ceroidlipofuscinosis type 2 (CLN2) disease. RGX-181demonstrated promising results, including reducedseizures (86% reduction), increased intervals betweenenzyme replacement therapy infusions, and improvedmotor and language skills after six months. RGX-181 was well tolerated with no serious adverse events.

CARDIOLOGY

BioCardia has completed anexternal review of its Phase III pivotal trial of CardiAMP CellTherapy for Heart Failure,which has FDA BreakthroughDesignation. The review confirmed the quality of the trial and its interim results. Although the interim analysis didn't include final results, it showed that the therapy was well tolerated and led to a reduction in all cause deaths and major adverse cardiac events. Though the results are clinically meaningful, they were notstatistically meaningful. BioCardia will continue toanalyze the data and work on improving efficacy

MESIAL TEMPORAL LOBE EPILEPSY (MTLE)

UniQure has received IND clearance for its gene therapy candidate, AMT-260, designed to treat refractory mesialtemporal lobe epilepsy (MTLE). AMT-260 utilizes an AAV9 vector to deliver engineered miRNAs targeting the GRIK2 gene, which is associated with seizures in refractory MTLE patients. A Phase I/IIa clinical trial,starting in Q4 2023, will assess safety, tolerability, and efficacy, offering hope for

MANUFACTURING

Cellares, a cell therapy manufacturer, secured $255 million in a series C investment round to finalize the constructionof its commercial-scale cell therapy manufacturing facility in Bridgewater, NJ. The total backing for the company is now $355 million. Cellares has introduced a novel acronym,IDMO (integrated development and manufacturingorganization), to describe its unique approach to cell therapy manufacturing, aiming to overcome the cost and scalability challenges faced by conventional CDMOs. The facility will be highly automated, utilizing a platform called ‘Cell Shuttles’, which allow for a tenfold increase in productivitycompared to traditional CDMO facilities. The facility will be 118,000 ft² and will produce 40,000 cell therapy batches annually. It is expected to be operational in the second half of 2024.