- | 9 Meters Biopharma
New Drug for Short-Bowel Syndrome Offers Hope of Simplified Treatment
Pharmacokinetics data on vurolenatide, a long-acting glucagon-like peptide-1 (GLP-1) being developed for short-bowel syndrome (SBS), suggest that the drug could be given twice monthly or less, an improvement over existing GLP-2 drugs, which must be given at least weekly.
- | ProQR
RNA-editing race intensifies as Big Pharma buys in
Dutch biotech firm ProQR Therapeutics announced it had struck its own RNA-editing partnership with Eli Lilly and Company to develop therapies for liver and nervous system diseases.
- | AltruBio
CMC Developability Assessments For Rapid Ph.1 Entry With AltruBio’s Gene Lee, Ph.D.
Fresh on the heels of an outstanding talk with AltruBio President & CEO Judy Chou, Ph.D., the company’s VP of Technical Development, Gene Lee, Ph.D., joins the Business of Biotech for a deep dive into the importance of CMC developability assessments. On this episode we cover the key points CMC developability assessments must address, when to begin them, how to go about them efficiently, and why they’re integral to early milestones and rapid entry into Phase 1 clinical trials.
- | Sio Gene Therapies
Sio Gene Therapies Touts Positive Safety and Biomarker Data for GM1 Gangliosidosis Candidate
On October 21st, Sio Gene Therapies Inc. made two major announcements featuring one of its lead gene therapy candidates. The New York-based company presented positive interim data for AXO-AAV-GM1, its AAV9-based gene therapy for GM1 gangliosidosis, at the ESGCT Virtual Congress 2021. On the same day, it also announced the FDA’s Fast Track Designation granted for the drug.
- | Cerevance
Cerevance seeks key to unlock brain disease treasure trove
Cerevance at Cambridge Science Park is on the cusp of discovering potential treatment options. Since inception, the company has collected more than 250 billion transcripts of data as part of research into degenerative brain diseases.
- | Sio Gene Therapies
ESGCT 2021 – Sio strengthens its case in GM1 gangliosidosis
Sio Gene Therapies had already reported decent results with a low dose of AXO-AAV-GM1, its gene therapy candidate for the rare inherited disease GM1 gangliosidosis. Now it has posted even more impressive data with a high dose of the project.
- | Selecta Biosciences
Gene Therapy Redosing Gains Traction as Need Grows and Novel Strategies Emerge
In fact, “approximately 95% of gene therapies today use adeno-associated viruses (AAVs) to deliver a transgene to its target. However, because the AAVs are highly immunogenic, you can’t give a second dose,” Carsten Brunn, Ph.D., president and CEO of Selecta Biosciences, told BioSpace. To further compound matters, Brunn added, “up to 40% of patients have antibodies to AAVs, so they are not eligible for gene therapy.” Their immune systems would clear the therapy before it could take effect.
- | X4 Pharmaceuticals
Pharmashots Interview: X4 Pharmaceuticals’ Paula Ragan Shares Insights on the Data of Mavorixafor in Combination with Ibrutinib Presented at 2021 EHA Annual Congress
In an interview with PharmaShots, Paula Ragan, Ph.D., President, and Chief Executive Officer at X4 Pharmaceuticals shared her views on preliminary efficacy and safety data of Mavorixafor + Ibrutinib in an ongoing P-Ib study to treat waldenström’s macroglobulinemia
