Passage Bio Inc.

Passage Bio's PBFT02 Shows Promise in Frontotemporal Dementia Trial

4 months ago

• Passage Bio's PBFT02 demonstrated a durable increase in cerebrospinal fluid progranulin (PGRN) levels in FTD-GRN patients during the Phase 1/2 upliFT-D trial. • Interim data showed a reduction in plasma neurofilament light chain (NfL) levels, a key biomarker, suggesting a potential therapeutic effect compared to natural history data. • The company plans to evaluate a lower dose (Dose 2) and anticipates reporting 12-month data from Dose 1, along with Dose 2 interim data, in the second half of 2025. • Passage Bio has completed process development for PBFT02, extended its cash runway into Q1 2027, and is on track to initiate dosing in FTD-C9orf72 patients in H1 2025.

Gene and Cell Therapies Show Promise in Treating CNS Disorders

6 months ago

• The market for gene and cell therapies targeting CNS disorders was valued at approximately USD 1 billion in 2021 and is projected to grow significantly by 2034. • Several companies are developing gene and cell therapies for CNS disorders, with some therapies already approved for conditions like spinal muscular atrophy (SMA). • Clinical trials are showing positive trends, such as uniQure's AMT-130 for Huntington's disease demonstrating encouraging results in mean CSF NfL levels.