Olivia Vizier

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

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 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.

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

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’ CHB portfolio includes STOPS (S-antigen transport-inhibiting oligonucleotide polymers), CAM (capsid assembly modulator), ASO (antisense oligonucleotide) and siRNA (small interfering RNA) candidates.

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.

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.

People with celiac most likely experience diarrhea due to intestinal inflammation and damage caused by gluten, says Patrick Griffin, MD, gastroenterology specialist and Chief Medical Officer of 9 Meters Biopharma.

When startup TFF Pharmaceuticals raised $14 million in a series A in 2018, its intention was to deploy its dry-powder drug-delivery technology toward making novel inhalable therapies for asthma, chronic obstructive pulmonary disease and other serious lung diseases. Then COVID-19 hit, and the company saw an opportunity to apply the platform to the emerging virus.

Don’t call Noubar Afeyan a venture capitalist. Sure, the 59-year-old founder and CEO of “venture creation” firm Flagship Pioneering has helped launch some 70 biotech companies in the past several decades. But Flagship both backs and performs the original scientific research of his portfolio companies. Its best-known launch is Moderna (ticker: MRNA), the vaccine-developer and

Selecta’s platform makes use of a biodegradable nanoparticle that encapsulates the immunosuppressive drug rapamycin, and is designed to be given in conjunction with gene therapies to induce antigen-specific immune tolerance.

Brad Margus, chief executive of drug-discovery company Cerevance, said that lowering drug prices will especially hurt companies working on the hardest-to-crack diseases, where even though a drug that works would have a huge market, it’s nearly impossible to persuade investors to support new approaches. “The price controls now being pushed threaten to remove the chance for

Celyad is focusing on advancing chimeric antigen receptor T cell (CAR T) therapies. “Approved cell therapy products have a very, very high response rate,” he said. “The challenge is in expanding that therapy from hematologic malignancies to other targets – in particular, to solid tumors – where we, as a field, haven’t yet cracked the

Dale Christensen is the Director of Clinical Development at TFF Pharmaceuticals. It’s an Austin-based company and specializes in “Thin Film Freezing”—a technology first created at UT Austin.

Elicio Therapeutics’ lymph node-targeting vaccine displayed cellular and humoral immunity to SARS-CoV-2 in a pre-clinical study.

Scynexis is introducing Brexafemme (ibrexafungerp tablets), the first and only oral non-azole prescription medication approved by the Food and Drug Administration to treat vulvovaginal candidiasis, commonly referred to as vaginal yeast infection.

In an interview with PharmaShots, James E. Brown, President, CEO, and Director of Durect shared his views on the clinical data of DUR-928 in P-1b trial for NASH and P-I trial for Hepatic Impairment

In an interview with PharmaShots, James Graham, CEO of Recce Pharmaceuticals shared his views on the multiple patient dosing in P-I/II clinical trial of RECCE 327 (R327) for the treatment of infected burn wounds.

AltruBio has signed a new chief medical officer, five months after a makeover that saw it change its name, its focus and its board.

Cardiff’s onvansertib is being trialed in a phase 1b/2 study in patients with metastatic colorectal cancer who have a mutation of the KRAS gene, according to a Wednesday release. The therapy is being combined with chemotherapy and Roche’s stalwart Avastin. Patients must have failed on chemotherapy and or/Avastin prior to entry in the trial.

Dorigo is principal investigator of a clinical trial of a new immunotherapy called maveropepimut-S. It’s being developed by IMV Inc., a pharmaceutical company focused on innovative immune cell activating approaches.

At the heart of Araris’ platform is its proprietary line of indication-specific peptide linkers with finely tuned hydrophilicity to accommodate various payloads, composed from an enzyme that’s already been approved by the FDA for food use. A native glycosylated antibody, the Araris linker with the payload, bind to form the ADC under the catalyzing effect

With its controversial Alzheimer’s med donanemab nearly ready for FDA scrutiny, Eli Lilly has grown more emboldened in its efforts to become the top dog in neuroscience. A new partnership with a quiet RNA editing player focused around neuroscience could now add even more bite to Lilly’s bark.

“It’s not exactly rocket science,” Ludek Sojka, CTO, SOTIO, admitted during our conversation on managing raw material supplier relationships to better control overall C&G therapy COGS. However, the past year has proven that securing a reliable supply of raw materials will — despite our best intentions — always be an evergreen challenge. A great reminder

What it means to age is changing, and William Greene, M.D., CEO of Fountain Therapeutics, is one of those driving the change. Rather than focusing primarily on lifespan, he and his company are focusing on healthspan, and in particular, the impact of chronic degenerative diseases. The goal is to restore cellular resilience.