GM1 gangliosidosis is a rare inherited disease with no FDA-approved treatments currently. Safety is a big concern in the field of AAV gene therapy and several promising endeavors have been shut down recently due to adverse events. However, AXO-AAV-GM1 has shown promising safety and efficacy in both the low-dose and high-dose cohorts.
"The results from the GM1 gangliosidosis program is one of the light bulb moments for me and I think this gene therapy could really make a difference," Pavan Cheruvu told GeneOnline. "With these important data in hand, we anticipate a meeting with the FDA in the first half of 2022 to discuss the next steps for clinical development for AXO-AAV-GM1."
The skin is colonized by bacteria and other microbes since our birth and the hormonal changes that occur during puberty are responsible for acne, one of the commonest skin problems. Belgian company S-Biomedic is currently researching how to best use the skin microbiome to treat acne and is confident to release a consumer product in the next couple of years.
Besides acne, companies are also evaluating their skin microbiome products for body odor, atopic dermatitis, or psoriasis. Apart from biotech companies, cosmetic companies are also working on microbiome-based solutions with Loreal working to add anti-dandruff shampoo, anti-blemish, and probiotic enriched anti-aging sera to its product lines. At this pace, a day at the spa with microbiome-perfected products may not be very far.
The first keynote panel discussion had industry experts look at the future of the healthcare innovation ecosystem in Europe. The panel comprised of 1. Nathalie Moll, Director General of EFPIA, European Federation of Pharmaceutical Industries and Associations 2. Jo De Cock, , Former Administrative General, INAMI, Institut national d'assurance maladie-invalidité, Belgium 3. Meindert Boysen, Director of National Institute of Health and Care Excellence, UK 4. Iskra Reic, Executive VP-AstraZeneca, Europe and Canada
Recounting their key learnings from the pandemic, the panelists shared optimism in incorporating collaboration and flexibility into building the future healthcare infrastructure.
Antibiotics developed to have broad-spectrum activities directly affect the gut microbiota, thereby compromising their multiple beneficial effects and weakening the body's first line of defense against pathogens. The most common side effects of this collateral damage are gastrointestinal problems and recurrent Clostridiodes difficle infections. This damage results in long-term health issues such as the development of allergic, metabolic, immunological, or inflammatory diseases.
Recently, EMBL researchers, along with their collaborators, analyzed the effects of 144 antibiotics on the most common gut microbes. The study published in Nature illuminates the activity spectra of antibiotics in commensal bacteria and suggests strategies to circumvent their adverse effects on the gut microbiota. This collaborative effort not only improves our understanding of the effects of antibiotics on gut microbes but also suggests a new approach to overcome them.