- | 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
- | Aligos Therapeutics
Aligos Therapeutics targeting functionally curative HBV regimen
Aligos’ CHB portfolio includes STOPS (S-antigen transport-inhibiting oligonucleotide polymers), CAM (capsid assembly modulator), ASO (antisense oligonucleotide) and siRNA (small interfering RNA) candidates.
- | Valneva
Valneva Says Its Covid Vaccine Beats AstraZeneca’s. Shares Are Surging.
American depositary receipts of Valneva jumped more than 30% Monday after the French biotech said that phase-3 trials of its Covid-19 vaccine VLA2001 had shown it triggered a stronger immune response and fewer side-effects than the shot of competitor AstraZeneca.
- | MeiraGTx
This Small Biotech Is Partnering With Johnson & Johnson. The Stock Could More Than Double.
Among them is MeiraGTx Holdings, shares of which fell from a 2019 peak of $30 to a recent $14, leaving its market capitalization below $600 million—and only a half-dozen analysts paying it heed.
