NOVARTIS

The global Retinal Gene Therapy market, valued at US$119.15 million in 2024, is projected to grow at a CAGR of 9.6% through 2034. Driven by advancements in gene therapy technologies and clinical successes, it offers hope for treating inherited retinal diseases like retinitis pigmentosa and Leber congenital amaurosis. Key players include Novartis AG and Spark Therapeutics, with recent developments focusing on expanding treatment options and manufacturing capabilities.

Novartis' HRO761, an oral allosteric WRN helicase inhibitor for MSI-high and dMMR tumors, was discovered through specific HTS assays and optimized for LipE, TPSA, chameleonicity, and non-classical zwitterions. Its X-ray structure and preclinical activity are detailed, differentiating it from Vividion's VVD-214.

FDA granted priority review to Novartis’ Scemblix for treating newly diagnosed Ph+ CML-CP patients, based on Phase III ASC4FIRST trial data showing superior efficacy and safety over standard TKIs. Scemblix demonstrated higher MMR rates, fewer adverse events, and aligns with previous safety profiles. Already approved for previously treated patients, it represents a potential new standard in CML treatment.

The FDA granted priority review to asciminib for treating newly diagnosed Ph+ CP-CML, supported by the ASC4FIRST study showing superior MMR rates over SOC TKIs. Asciminib demonstrated a 67.7% MMR rate vs 49.0% for SOC TKIs, with fewer AEs. It received breakthrough therapy designation and is under FDA's Real-Time Oncology Review.

The "Hypoglycemia - Global Clinical Trials Review, 2024" report offers an overview of global hypoglycemia clinical trials, detailing trial numbers, enrollment, and trends by region, country, phase, status, and sponsor type. It highlights key companies and drugs involved, aiding in strategic investment and identifying clinical trial opportunities.

The global Retinal Gene Therapy market, valued at US$119.15 million in 2024, is projected to grow at a CAGR of 9.6% through 2034. Driven by advancements in gene therapy technologies and clinical successes, it offers hope for treating inherited retinal diseases. Opportunities include diversifying targeted diseases and leveraging gene editing technologies like CRISPR-Cas9. Major players include Novartis AG and Spark Therapeutics, with recent developments highlighting strategic collaborations and manufacturing expansions.

In 2024, global drugmakers like AstraZeneca and Novartis are pursuing deals in China to enhance their drug pipelines and market presence, despite Sino-US tensions. This interest benefits local companies and investors, though risks remain due to political and economic uncertainties. Foreign acquisitions in China's healthcare sector have decreased, with domestic buyers dominating.

Global pharmaceutical companies are increasingly targeting China's expanding healthcare market, driven by its aging population and rising chronic disease rates. Despite geopolitical tensions and regulatory challenges, major deals like AstraZeneca's $1.2 billion acquisition of Gracell Biotechnologies highlight the market's appeal. Investments in local R&D and strategic partnerships are key strategies for navigating China's complex landscape, aiming to introduce innovative treatments and strengthen market presence.

Global drugmakers like Bristol Myers Squibb and Sanofi are pursuing M&A deals in China to enhance drug pipelines and market presence, despite Sino-U.S. tensions and regulatory challenges. Recent deals include AstraZeneca's $1.2B purchase of Gracell Biotechnologies. Political and economic risks, including U.S.-China relations and data transfer restrictions, are significant considerations.

Gene therapy involves modifying genetic material to treat diseases, with mRNA and genome-editing technologies like ZFNs, TALENs, and CRISPR/Cas9 showing promise. Despite advancements, challenges include delivery efficiency, safety, and immune responses. mRNA therapy offers rapid production and avoids genome integration but faces stability and delivery hurdles. Genome-editing tools enable precise genetic modifications, with CRISPR/Cas9 leading in efficiency and application, though off-target effects and delivery systems remain concerns. AAV and LNP are key delivery systems, each with advantages and limitations in gene therapy applications.