This week we highlight potential new medtech regulations in the UK, challenges facing CAR-T therapies and another CRISPR-based gene therapy approval. We also examine the multi-faceted approach being taken by biotech companies seeking funding or potential exits. Additionally, we look at a new device for skin cancer screening.
Each week we highlight five things affecting the life sciences industry. Here’s the latest.
UK regulator announces significant changes for medtech companies
The U.K. Medicines and Healthcare products Regulatory Agency has outlined a two-year roadmap to create a new framework for the medtech sector, according to Medtech Dive. The agency aims to discuss changes in areas such as unique device identification and quality management systems with key industry stakeholders, with plans to establish the “core elements” of the new framework by next year. The agency is also interested in improving international recognition by facilitating swifter access to devices approved by comparable regulators.
CAR-T therapies face significant hurdles
CAR-T therapies, which emerged in the last decade to treat blood cancers, among other indications, have faced significant roadblocks, according to Fierce Pharma. Among these are manufacturing challenges, criticism of pricing, provider capacity, and the need for designated treatment centers due to complex logistics. While there is optimism about the efficacy and revenue potential of CAR-T therapies, the limitations of health care infrastructure, including hospital capacity and staffing, may impact their widespread adoption. Efforts are underway to address these challenges, but the slow pace of organizational change and infrastructure development poses long-term considerations for the CAR-T landscape.
New CRISPR-based gene therapy receives second FDA approval
Recently, the U.S. Food and Drug Administration has granted approval for Casgevy, the first CRISPR-based medicine, to treat sickle cell disease, as reported in U.S. News & World Report. This week, the drug received a second approval for the treatment of transfusion-dependent beta-thalassemia, an inherited blood disorder. The therapy involves a one-time dose that permanently alters DNA in a patient’s blood cells using CRISPR/Cas9 technology. Despite its high list price of $2.1 million, the drug addresses severe indications for which there are limited treatment options.
Biotech companies take multifaceted approach to prepare for potential fundraising or exit events
More than ever, biotech companies are increasingly adopting a dual-track strategy, pursuing both initial public offerings and acquisitions to maximize their potential, according to a Fierce Biotech article. This approach, considered a necessity in the competitive and dynamic biotech market, allows companies to remain flexible and adapt to changing circumstances. The multi-track process involves significant resources, including legal, auditing and banking support, and companies weigh the options of IPO funding, licensing, or mergers and acquisitions based on what proves more compelling for investors. Executives in the industry understand the need to explore all potential fundraising and exit scenarios given some cautious optimism about a potential opening in the capital markets and recent increases in licensing and acquisitions from large pharmaceutical companies.
FDA approves AI-powered device to detect skin cancer
The FDA has granted clearance for a new artificial intelligence-powered handheld probe that aids in identifying skin cancer signs in moles, bumps or lesions, according to Fierce Biotech. Designed for use by primary care providers, the noninvasive optical spectroscopy system assists in recognizing the three most common skin cancers (melanoma, basal cell carcinoma and squamous cell carcinoma) at the point of care for individuals aged 40 and above. While not a standalone diagnostic tool, the product offers immediate results, guiding physicians to either “investigate further” or “monitor,” addressing a critical need in medicine for early skin cancer detection.
