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Message : Required fields Passage Bio collaborator University of Pennsylvania’s Gene Therapy Program to present poster regarding development of a novel mouse model for evaluating efficacy of adeno-associated virus gene therapy for metachromatic leukodystrophy (MLD) Passage Bio gene therapy candidate, PBML04, showed preliminary signs of efficacy in novel mouse model of MLD PHILADELPHIA, May 11, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical-stage genetic medicines company focused on developing transformative therapies for rare monogenic central nervous system (CNS) d...

Published: May 10, 2021 Galafold® (migalastat) PerformanceReflects Continued Strong Adoption in All Key Global Regions; On-Track to Achieve Revenue Guidance of $300M-$315M 1Q21 Total Galafold Revenue of $66.4M Driven by Continued Global Growth Positive Pre-BLA Meeting Held with U.S. FDA for AT-GAA in Pompe Disease; Rolling BLA Submission On-Track for Completion in 2Q21with Global Submissions Expected Throughout 2021 New Data from Pompe and Fabry Gene Therapy Programs to be Presented at American Society of Gene & Cell Therapy 24th Annual Meeting May 11th-14th Continue to Strengthen Senior...

Published: May 06, 2021 SOUTH SAN FRANCISCO, Calif., May 06, 2021 (GLOBE NEWSWIRE) -- Catalyst Biosciences, Inc.. (NASDAQ: CBIO) today announced its operating and financial results for the first quarter ended March 31, 2021 and provided a corporate update. “We made significant progress across our Protease Medicines platform, specifically in our complement and hemostasis programs. We are preparing to initiate an observational trial in patients who have diseases related to CFI deficiency in mid-year 2021 to support our SQ enhanced CFI development candidate CB 4332 that will enter the clin...

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Better Outfitted and Screened Viral Vectors Are Safer and More Effective Better Outfitted and Screened Viral Vectors Are Safer and More Effective Adeno-associated virus (AAV) vectors are the leading vectors for gene delivery. Two AAV-based gene therapies have been approved by the U.S. Food and Drug administration, and dozens more are in clinical trials. However, AAV vectors pose challenges including costly manufacture, high percentages of empty capsids, and safety issues associated with high-dose regimens. To address this last challenge, developers are working to improve the impact of each A...