Moderna

EU authorities to decide on Sputnik V vaccine use in September; Bavarian PM Markus Söder urges EMA for swift approval. Despite EMA's ongoing review, Hungary and Slovakia use Sputnik V without its permission.

The North America RNA Therapeutics Market is projected to grow from USD 12.95 billion in 2023 to USD 17.79 billion by 2029, driven by advancements in RNA technology and personalized medicine. Key players include Moderna, Alnylam Pharmaceuticals, and Pfizer Inc.

Meiji Seika Pharma invests in ARCALIS to enhance mRNA vaccine production in Japan, aiming to address COVID-19 vaccine delays. The partnership aims to establish a comprehensive domestic mRNA vaccine production system by December 2024, aligning with the Japanese government's vaccine strategy.

Valo Health appoints Brian M. Alexander as CEO, Brett Blackman as COO, and Travis Wilson to its Board of Directors, leveraging AI and data for drug discovery.

Novavax received FDA approval to resume clinical trials for its Covid-19 and influenza combination vaccine (CIC) and standalone influenza vaccines after a month-long hold. The Phase III trial will evaluate the CIC vaccine's efficacy in adults aged 65 and older, comparing its immune response to standard flu and Covid-19 vaccines. Novavax aims to commence enrolment by the end of 2024, with preliminary results expected by mid-2025.

Cell and gene-based therapies targeting RNA or DNA offer potential for treating rare genetic diseases, but commercial incentives often fail to support the rarest conditions. Recent examples show genetically-targeted technologies can develop individualized therapies for unique genetic pathologies, applicable to small patient markets. FDA guidances govern individualized therapies, but specialized trial design and clinical outcome measures are needed. N-of-1 trials for individualized, genetically targeted therapies face challenges in clinical protocol development and defining meaningful changes. Clinical outcome assessments should reflect patient's lived experience, and safety measures must match potential toxicity profiles. Future statistical and regulatory considerations are crucial for advancing these therapies.

Novavax received FDA approval to resume clinical trials for its Covid-19 and influenza combination vaccine (CIC) and standalone influenza vaccines after a month-long hold. The Phase III trial will evaluate the CIC vaccine's efficacy in adults aged 65 and older, comparing its immune response to standard flu and Covid-19 vaccines. Novavax aims to commence enrolment by the end of 2024 with preliminary results by mid-2025.

The FDA's approval of the first CRISPR therapeutic, Casgevy, marks a milestone in gene-editing therapies, with over 300 CRISPR-based treatments in development. Innovations in drug-delivery vectors, such as nonviral options like lipid nanoparticles (LNPs), polymer-based nanoparticles, and extracellular vesicles (EVs), are crucial for enhancing specificity, efficiency, and safety. These advancements could reduce healthcare costs and improve patient access, potentially leading to cures for chronic diseases. Flow cytometry and analytical ultracentrifugation are key tools in vector development, aiding in the characterization and optimization of these delivery systems.

Novavax's COVID-19-influenza combination vaccine and stand-alone influenza vaccine candidates have had their clinical hold removed by the FDA after addressing safety concerns. The serious adverse event initially reported was unrelated to the vaccine, allowing for plans to resume Phase 3 trials.

Investors should focus on healthcare firms with strong business models, not solely on drug pipelines. Valuations should reflect core business strength, not speculative pipeline drugs. Opportunities exist in diagnostics, minimally invasive therapies, and telemedicine. Active management can identify companies with higher clinical success likelihood and strong R&D records.