Promising developments in gene therapy
Gene Tx offers novel treatments for complex diseases (680 words, 3.5 minutes)
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Gene therapy represents a groundbreaking approach that could offer promising solutions for genetic disorders and diseases. With advances in technology and the current understanding of the human genome, gene therapy has emerged as a powerful tool to address previously untreatable conditions at their root cause.
Speaking on Season 10 of the NPC Podcast, Brian Bloom, CEO and co-founder of Bloom Burton & Co., said that gene therapy is one of the major trends in the biotech industry. “Beyond using AI and computers to better make new drugs, the other major trend in the biotech industry is genetic medicines and the number of new modalities that we have to translate an idea or a new insight into a disease into what a therapy can be,” he said.
He emphasized it is possible to acquire a genetic understanding of diseases and create genetic medicines using techniques such as gene editing. He said RNA vaccines and RNA therapies based on RNA chemistry are examples. “We have all these new tools which only came around recently,” he said. “They were only dreamed up and thought of as possible maybe 20 years ago, and they just became a reality in the past 10 years, and everything is accelerating.”
According to research, adeno-associated virus (AAV) vectors are a significant development in gene therapy. These vectors have been effective in delivering therapeutic genes to targeted cells. Other gene-editing technologies, such as CRISPR-Cas9, allow for precise modifications to specific genes, offering potential treatments for diseases with identifiable genetic causes. Researchers have even used CRISPR to modify gene mutations linked to conditions such as specific types of cancer.
Advancements in gene therapy hold the potential to transform healthcare. Plenty of companies around the world are working to further develop the field, including Ambry Genetics. Speaking on the first episode of Season Nine of the NPC Podcast, Danielle Portnik, Regional Business Director, International at Ambry Genetics, said the company translates scientific research into something clinically actionable in genetic test results. “This comes from a really deep understanding of the human genome, the biology behind genetic disease, and what we’re doing to move that into the future,” she said.
Sources:
https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
https://medicine.missouri.edu/centers-institutes-labs/health-ethics/faq/gene-therapy
https://www.cancer.gov/news-events/cancer-currents-blog/2020/crispr-cancer-research-treatment
THIS WEEK 06/27/23
Takeda Canada announced the Institut national d'excellence en santé et en services sociaux (INESS) recommended maribavir (Livtencity) for public reimbursement in Quebec. Livtencity is indicated for the treatment of adult patients with post-transplant cytomegalovirus disease.
Health Canada approved Sun Pharma’s isotretinoin capsules (prAbsorbica LD) for the treatment of severe nodular acne in patients 12 years of age and older.
Agepha Pharma announced the U.S. FDA has granted approval to colchicine (Lodoco), an anti-inflammatory atheroprotective cardiovascular drug that reduces the risk of myocardial infarction, stroke, coronary revascularization, and cardiovascular death in adult patients.
The U.S. FDA approved Sarepta Therapeutics’ delandistrogene moxeparvovec-rokl (Elevidys), an adeno-associated virus based gene therapy, for the treatment of ambulatory pediatric patients between four and five years with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene.
LISTEN UP
In season 10 of the NPC Podcast, Justin Bates, CEO of the Ontario Pharmacists Association, discusses starting his position just before Covid, shifting pharmacies to a service model, and alternative payment models to National Pharmacare. Hear him in conversation with podcast hosts Mitch Shannon, Jim Shea and Mark McElwain.
HEALTHBIZ REWIND
This feature of NPC Healthbiz Weekly looks back at some of the most insightful moments from previous NPC Podcast episodes. In Healthbiz Rewind, you’ll read bold life sci predictions made during the renowned “Prognostication Korner” segment of the NPC Podcast.
Arnaud Lavenue
Senior Partner
Toc Toc Communications
Montreal
Season 9, episode 02
Listen to this episode here
What bold predictions will you make about the life sciences industry during the coming 12 to 24 months?
Maybe this is some wishful thinking, but I anticipate some bold moves. One [bold move] would be turning the overwhelming data collection that we have into easy access to a real-world communication platform that responds to the needs of the different stakeholders. I'm talking about physicians, the medical associations, the patients, the authorities, the patient associations, etc. It's a big challenge, but with potentially significant and positive outcomes in terms of transparency and learning, but also in terms of turning insights into new actions for the benefit of both physicians and patients.
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