NICE Recommends Novel Gene Therapy for Rare Genetic Brain Disorder
The UK’s National Institute for Health and Care Excellence (NICE) has issued a draft guidance recommending the approval of Upstaza, PTC Therapeutic’s gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. The gene therapy, if approved, would cost £3 million and would be the only treatment for the ultra-rare disorder.
Making Next-Gen Sequencing Accessible to Patients
Next-generation sequencing (NGS) is essential for advances in individualized medicine, but getting it to patients requires convincing insurers to pay for the technique. For this goal, care must be taken to ensure an accurate value-assessment process. In a new article in Value and Outcomes Spotlight, Lieven J.P. Annemans, PhD, discusses the barriers patients face accessing NGS and proposes a plan of action to reduce them.
Cell & Gene Day 2022 | Virtual Event | August 24th
Cell and gene therapies are set to explode over the next decade. However, reaching their full potential will take careful consideration to avoid the pitfalls in drug development, manufacturing, and market access. Tomorrow, August 24th, Endpoints News and Precision ADVANCE will host Cell & Gene Day, featuring a stellar lineup of talks from industry leaders. The first of three panels is entitled, “Future technologies for cell & gene therapy.”
ICER Report At a Glance: Beti-Cel for Beta Thalassemia
Beta Thalassemia is a blood disorder that results in dangerously low levels of functional hemoglobin and requires expensive lifelong medical care, including regular blood transfusions. Beti-cel is a gene therapy by Bluebird Bio that can potentially cure the disorder by genetically modifying a patient's own hematopoietic stem cells (HSCs). The Institute for Clinical and Economic Review (ICER) has just released its assessment of Beti-cel, finding that it is effective and could save money on treatment, but more comprehensive long-term data would be necessary to determine the degree of the net health benefit over the current standard of care.
Syndicated Report: Global Gene Therapy Market Projections 2022-2031
Despite the global pandemic, cancer was still the leading cause of death in 2020. This pressing therapeutic need underlies the growth projected by Kenneth Research in their new report entitled, “Global Gene Therapy Market.” The report predicts that the gene therapy market will earn around $6 billion USD from 2022-2031 with a CAGR of ~34%.
Carbon Bioscience Partners with the Cystic Fibrosis Foundation to Develop New Viral Vector-Based Therapy
Although adeno-associated virus (AAV) gene therapies have represented a large swath of the gene therapeutic landscape for years, they have [...]
CMC – Gene Therapy’s “Achilles Heel”
Patient access to novel gene therapies is often obstructed by manufacturing challenges, in particular, issues with Chemical, Manufacturing and Controls [...]
On-Demand Webinar: Navigating the Path to the Clinic And Beyond For Gene Therapies
Bringing novel gene therapies from the drawing board to patients requires careful consideration of each step of the pipeline. A [...]
Webinar: Learning from the Pandemic to Accelerate AAV-based Gene Therapies
On Thursday, March 17th, Fierce Biotech and Cytiva present a webinar on applying lessons from the COVID-19 pandemic to accelerate [...]
On-Demand CE Course on Effective Novel Gene Therapies in Hemophilia; Implementing Value-Based Coverage Policies
Novel gene therapies are of particular interest in treating hemophilia. Despite promises of efficacy and even curing the disease for [...]
Managed Entry Agreements Could Bring Affordability to Multibillion-Dollar Novel Gene Therapy Market
Cogentia’s Senior Analyst Mark Orchard discusses how managed entry agreements (MEAs) could become a major trend in improving access to [...]
