Comment
Author: Admin | 2025-04-28
Systemic 2e13 36.9% 9/54 Under regulatory review Dimension Therapeutics (DTX101) Codon-optimized FIX 96 AAVrh10 ssAAV Systemic 1.6e12-5e12 6.7% 3/3 at highest dose Closed Freeline Therapeutics (FLT-180a, verbrinacogene setparvovec) Codon-optimized FIX containing the Padua mutation 5 AAV-S3 synthetic capsid ssAAV Systemic 3.84e11-1.28e12 30%-279% 8/10 Clinical trials ongoing Belief BioMed (BBM-H901) Codon-optimized FIX containing the Padua mutation 0 AAV843 synthetic capsid scAAV Systemic 5e12 36.9% 2/10 Clinical trials ongoing Sangamo Bioscience (SB-FIX) Codon-optimized FIX Not known AAV6/zinc finger–mediated targeted integration into the albumin locus in hepatocytes ssAAV Systemic Unknown Unknown Closed SponsorTransgeneNo. of CpG motifs in transgeneSerotypeGenome formatMethod of vector deliveryDose range (vg/kg)Mean stable FIX activity levelsNo. of patients with transaminitisCurrent statusAvigen/CHOP Wild-type FIX 19 AAV2 ssAAV IM 2e11 to 1.8e12 Transient at a maximum level of 1.6% 0 Closed Avigen/CHOPCoagulin-B Wild-type FIX 19 AAV2 ssAAV Intrahepatic artery 2e11 to 2e12 Transient with a maximum of 12% 1/2 at highest dose Closed St Jude/UCL Codon-optimized FIX 0 AAV8 scAAV Systemic 2e11 to 2e12 5.1% 4/6 patient at highest dose Closed Takeda (Baxalta; BAX 335) Codon-optimized FIX + Padua mutation 99 AAV8 scAAV Systemic 2e11 to 3e12 Transient except in 1 patient who had expression of ~20% at last report 2/6 patients treated at or above 1e12 received steroids in response to ALT elevations and 1 patient received prophylactic steroids. Closed Spark Therapeutics (SPK-9001, fidanacogene elaparvovec) Codon-optimized FIX containing the Padua mutation 0 AAV- Spark100 ssAAV Systemic 5e11 19.8% at 5 years 3/15 patients Transitioned to phase 3 with Pfizer uniQure (AMT-060) Codon-optimized FIX 0 AAV5 ssAAV Systemic 5e12-2e13 6.9%5.2% at 5e12-vg/kg dose and 7.2% at 2e13-vg/kg dose at 5 years 2/5 at highest dose A new program, AMT-061, that contains the FIX Padua in development with CSL Behring CSL Behring (AMT-061, etranacogene dezaparvovec) Codon-optimized FIX containing the Padua mutation ?0 AAV5 ?ssAAV Systemic 2e13 36.9% 9/54 Under regulatory review Dimension Therapeutics (DTX101) Codon-optimized FIX 96 AAVrh10 ssAAV Systemic 1.6e12-5e12 6.7% 3/3 at highest dose Closed Freeline Therapeutics (FLT-180a, verbrinacogene setparvovec) Codon-optimized FIX containing the Padua mutation 5 AAV-S3 synthetic capsid ssAAV Systemic 3.84e11-1.28e12 30%-279% 8/10 Clinical trials ongoing Belief BioMed (BBM-H901) Codon-optimized
Add Comment