PTC Therapeutics said on Monday, September 21, 2026 that it completed its acquisition of ST-920 (isaralgagene civaparvovec) from Sangamo Therapeutics. ST-920 is a one-time AAV gene therapy for Fabry disease that is designed to enable long-term production of the missing alpha-galactosidase A enzyme in place of chronic enzyme replacement therapy. Under terms announced in August, PTC won the asset in Sangamo's Chapter 11 auction for $111 million upfront plus up to $100 million in approval milestones; Eli Lilly separately bought Sangamo's zinc finger programs for $50 million. ST-920 holds FDA Orphan Drug, Fast Track, and RMAT designations and EMA PRIME eligibility, and PTC expects to complete a rolling Biologics License Application for accelerated approval in the fourth quarter of 2026.
uniQure N.V. (NASDAQ: QURE) announced Wednesday September 2, 2026 the submission of a Biologics License Application (BLA) to the U.S. FDA for the accelerated approval of ifezuntirgene inilparvovec (AMT-130), an investigational AAV-delivered gene therapy for the treatment of Huntington's disease. The BLA is the first ever submitted for a Huntington's disease therapy of any kind. AMT-130 is delivered by stereotactic intraparenchymal injection into the striatum and expresses a microRNA designed to silence the mutant huntingtin (mHTT) gene. The submission is supported by three-year data from the Phase 1/2 program compared to a propensity score-matched external control from the Enroll-HD natural history database. uniQure also submitted a Marketing Authorisation Application (MAA) to the UK MHRA on the same day. A four-year data readout from the ongoing Phase 1/2 study is expected before end of Q3 2026. AMT-130 previously received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA. Approval would establish the first disease-modifying therapy for Huntington's, where standard-of-care remains symptom management with tetrabenazine and deutetrabenazine plus antipsychotics.
Regenxbio (NASDAQ: RGNX) shares fell approximately 25% to $8.05 Tuesday August 25, 2026 after the FDA placed a clinical hold on the RGX-121 gene therapy program for the treatment of Mucopolysaccharidosis type II (MPS II, also known as Hunter Syndrome). Disease context: MPS II is a rare X-linked lysosomal storage disease affecting approximately 1 in 100,000-170,000 male births globally, caused by iduronate-2-sulfatase (IDS) enzyme deficiency that results in accumulation of glycosaminoglycans (GAGs) in multiple tissues including the central nervous system. Clinical manifestations include progressive cognitive decline, skeletal abnormalities, cardiac and respiratory involvement, and reduced life expectancy in the severe neuronopathic form (approximately two-thirds of patients). RGX-121 mechanism: an AAV (adeno-associated virus) gene therapy delivering an IDS-encoding transgene via intracerebroventricular administration designed to establish stable IDS expression in the central nervous system, addressing the neurocognitive manifestations that the currently-approved enzyme replacement therapy Elaprase (idursulfase, Takeda) does not reach because Elaprase administered intravenously does not cross the blood-brain barrier. Specific reasons for the FDA clinical hold have not been publicly disclosed by Regenxbio; details are expected in subsequent regulatory correspondence. The clinical hold delays but does not necessarily preclude the RGX-121 development pathway. Regenxbio's broader pipeline includes RGX-202 for Duchenne muscular dystrophy in Phase 3, RGX-314 for wet AMD in Phase 3, and multiple additional gene therapy candidates.
Voyager Therapeutics (NASDAQ: VYGR) presented six-month Good Laboratory Practice (GLP) toxicology data for VY1706, its investigational blood-brain-barrier-crossing AAV gene therapy targeting tau for Alzheimer's disease, in a Developing Topics late-breaking poster at AAIC 2026 in London on Monday July 13, 2026. The updated six-month data extends the prior three-month timepoint (64% reduction reported earlier this month): a single intravenous dose delivered sustained tau protein reduction up to 75% in key brain regions of non-human primates over six months. VY1706 was well tolerated with no adverse clinical pathology and no histopathological findings up to the highest dose tested. Notably, the program showed none of the typical AAV liver transaminase elevations at any dose level throughout six months, plasma neurofilament levels remained generally stable with no dose-related increases, and there were no cellular immune activations. Voyager received FDA Investigational New Drug (IND) clearance for VY1706, enabling initiation of a clinical trial in adults with early Alzheimer's disease with dosing expected in the second half of 2026.
Voyager Therapeutics (NASDAQ: VYGR) presented Developing Topics late-breaking poster data at AAIC 2026 in London (July 12-15) featuring VY1706, its investigational blood-brain-barrier-crossing AAV gene therapy targeting intracellular and extracellular tau for Alzheimer's disease. The 3-month GLP non-human primate toxicology study showed VY1706 was well tolerated at a single intravenous dose up to the highest level tested (5E13 vg/kg), with no adverse clinical pathology or histopathological findings. Tau protein was reduced up to 64% in key brain regions of non-human primates at 13 weeks following a single IV dose. Voyager received FDA Investigational New Drug (IND) clearance for VY1706 in H1 2026, enabling initiation of a clinical trial in adults with early Alzheimer's disease with dosing expected in the second half of 2026. The program extends the same BBB-delivery-plus-tau-reduction thesis that Denali's DNL628 OTV pursues via anti-tau ASO; both address the same therapeutic hypothesis through different modalities (AAV gene therapy vs. anti-sense oligonucleotide).