Vertex Pharmaceuticals is known for its cystic fibrosis drugs, but the company aims to diversify with a pipeline that reaches more therapeutic areas. CEO Reshma Kewalramani explained Vertex’s strategy during the World Medical Innovation Forum in Boston.
Vertex Pharmaceuticals gains a Phase 3-ready Alpine Immune Sciences drug with potential applications in a wide range of immunological disorders. The lead indication for the Alpine drug, povetacicept, is the chronic kidney disease IgA nephropathy.
As technology advances, AI-powered tools will increasingly reduce the administrative burdens on healthcare providers.
The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.
The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.
Vertex Pharmaceuticals aims to use CRISPR Therapeutics’ gene-editing technology to develop type 1 diabetes cell therapies that don’t prompt the immune system to reject them. This approach could compete with a Sana Biotechnology program expected to reach its first test in humans this year.
The future looks bright for the integrated specialty pharmacy model – for health systems, providers, and most importantly, for patients.
Vertex Pharmaceuticals already has a cell therapy candidate for type 1 diabetes but the company says acquiring rival ViaCyte will provide it with complementary assets and technologies. The two companies already share a partner in common: both have therapeutic candidates that use the gene-editing capabilities of CRISPR Therapeutics.
Vertex Pharmaceuticals is collaborating with Obsidian Therapeutics to develop up to five new gene editing therapies. Obsidian’s technology precisely controls protein expression, a capability the partners plan to apply to several undisclosed serious diseases.
Vertex Pharmaceuticals is amending its collaboration with CRISPR Therapeutics, paying $900 million now for chance to grab a bigger share of a gene-editing therapy’s profits later---if the treatment is approved. Clinical trials evaluating the CRISPR-based therapy are underway in sickle cell disease and beta thalassemia.
Phase III data on bluebird bio's Zynteglo, as well as Vertex and CRISPR Therapeutics' CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
The progress in artificial intelligence (AI) is reshaping patient care, across various dimensions, from facilitating faster discharge to curating treatment plans and suggesting lifestyle changes.
Analyst reports over the past few weeks have looked into what effects the Covid-19 pandemic could have on drugmakers' sales, examining multiple factors. But virtual launches and telemedicine could mitigate some market challenges.
The company, part of BioSig Technologies, aims to run a Phase II study of the drug, merimepodib, which had previously failed in a Phase IIb HCV study run by Vertex Pharmaceuticals. The company hopes the drug's mechanism could prove effective against SARS-CoV-2.
A recruiter in the industry said trial-monitoring staff report having their hours cut, while CRAs are grounded, and others have reported being laid off on social media. Several drugmakers have wound down clinical trial operations as the pandemic has overwhelmed a growing number of hospitals.
The biotech company said it is well-stocked to supply its approved drugs for cystic fibrosis to patients. Meanwhile, it plans to implement virtual visits and ship drugs to participants in its ongoing trials, while pausing enrollment and delaying initiation in some.
Shares of the company rose 9 percent on the news that the FDA had approved Trikafta. The drug will be an option for patients using the company's other approved CF drugs.