Historic market growth has been ... rare disease research pipelines, and the growing regulatory complexity associated with advanced therapies. Increased reliance on external scientific, clinical, and regulatory expertise has also strengthened demand for specialized contract research services. The cell and gene therapy CRO market is forecast to reach $12.36 billion by 2030, expanding at a CAGR of 12.3%. Key growth drivers include increasing clinical trial activity in ... Historic market growth has been supported by the rising number of early-stage cell and gene therapy programs, limited in-house development capabilities among therapy sponsors, expanding rare disease research pipelines, and the growing regulatory complexity associated with advanced therapies. Increased reliance on external scientific, clinical, and regulatory expertise has also strengthened demand for specialized contract research services. The cell and gene therapy CRO market is forecast to reach $12.36 billion by 2030, expanding at a CAGR of 12.3%. Key growth drivers include increasing clinical trial activity in gene therapy, greater investment in oncology and genetic disorder research, expansion of personalized medicine programs, and demand for faster development timelines. Regulators and healthcare systems are prioritizing precision-based treatments designed around genetic, molecular, and cellular characteristics, particularly for complex diseases with limited therapeutic options. Cell and gene therapy CROs support these programs through specialized preclinical studies, clinical trial management, regulatory strategy, safety monitoring, quality management, and analytical testing. Boost ROI by outsourcing R&D, integrating preclinical and clinical services, leveraging data-driven trials, strengthening compliance, and localizing labs... It also enhanced the organization's ability to manage global clinical trial programs involving advanced cell and gene therapies. The market supports a broad range of therapeutic areas, including oncology, central nervous system disorders, infectious diseases, immunological conditions, cardiovascular diseases, respiratory diseases, diabetes, ophthalmology, and pain management.

The pharmaceutical and technology group Merck is expanding its rare disease drug business through a billion-dollar acquisition. The DAX-listed company is buying British biotech group Silence... The pharmaceutical and technology group Merck is expanding its rare disease drug business through a billion-dollar acquisition. The DAX-listed company is buying British biotech group Silence Therapeutics for around $1.3bn, Merck said Monday. Merck is offering shareholders of the company, which is listed on the Nasdaq technology exchange in the United States, $20 in cash per American depositary share (ADS). Net sales break down by family of products as follows: - solutions and products for scientific and biotechnological research (42.6%); - pharmaceutical products (40.8%): prescription drugs for the treatment of diabetes, cancer, multiple sclerosis, infertility, cardiovascular disease, central nervous system disorders, inflammatory disorders, etc.; - performance materials for the electronics industry (16.6%): semiconductor materials, liquid crystals, pigments and additives, carbon-based organic materials, etc. This super composite rating is the result of a weighted average of the rankings based on the following ratings: Fundamentals (Composite), Valuation (Composite), EPS Revisions (1 year), and Visibility (Composite). This composite rating is the result of an average of the rankings based on the following ratings: Fundamentals (Composite), Valuation (Composite), Financial Estimates Revisions (Composite), Consensus (Composite) and Visibility (Composite). The company must be covered by at least 4 of these 5 ratings for the calculation to be carried out.

City Therapeutics’ lead RNA interference therapy could become an alternative to blockbuster-selling anticoagulants. The company’s technology for designing RNAi drugs and delivering them to tissues throughout the body has already led to partnerships with Bausch + Lomb in eye diseases and ... City Therapeutics’ lead RNA interference therapy could become an alternative to blockbuster-selling anticoagulants. The company’s technology for designing RNAi drugs and delivering them to tissues throughout the body has already led to partnerships with Bausch + Lomb in eye diseases and Biogen in central nervous system disorders. Using RNA to interfere with a disease-driving protein is a proven way to address disease targets in the liver. City Therapeutics is one of the companies developing the next-generation of these medicines for a wealth of targets in the rest of the body. With its lead drug candidate in clinical development for cardiovascular indications, the biotech now has $200 million in IPO cash to fund clinical development of that program and others in its pipeline. City is also exploring use of this siRNA for anemia associated with chronic kidney disease and chronic inflammatory disorders, including inflammatory bowel disease. In these indications, elevated hepcidin and iron restriction contribute to the disease. City’s research has attracted collaborators. Bausch + Lomb is working with the biotech to develop RNAi therapies for eye diseases, specifically geographic atrophy. Cambridge, Massachusetts-based City is developing RNA interference (RNAi) drugs, which employ small interfering RNA (siRNA) to target and degrade messenger RNA before it can be translated into a protein that contributes to disease. This modality, also called gene silencing, was pioneered by Alnylam Pharmaceuticals, which sells four FDA-approved RNAi drugs — all for rare diseases stemming from liver proteins.

So did investment firms, smaller biotechs, law firms, and banks. With Big Pharma firms readying to buy new assets in the face of looming patent cliffs, all kinds of partnerships are fair game: licensing deals, co-development agreements, and outright acquisitions. It’s not unusual for the firm’s partnerships to span a decade or more. “I think we really want to work with them and bring something forward, rather than buy a baked cake later,” he said. Boehringer on Tuesday announced a new collaboration with the antibody firm ComboTope Therapeutics to develop cancer drugs. Sanofi, the French pharmaceutical giant, is looking for deals in the immunology, rare disease, and vaccine spaces, according to Brian Bronk, head of business development, who spoke on a panel about dealmaking on Tuesday. The law subjects outgoing US investments to reviews by the US Departments of Treasury and Defense. If passed, the updated legislation would likely dampen the spate of US-China biotech deals, which recently include a $15.2 billion co-development agreement between Bristol Myers Squibb and Hengrui and a $10.5 billion partnership between Pfizer and Innovent. Maha Radhakrishnan, executive partner for private equity at Sofinnova Partners, told C&EN that she is interested in drug candidates for cancer, immunology and inflammation, cardiometabolic and renal diseases, central nervous system disorders, and rare diseases. San Diego—Pharmaceutical executives and biotech investors spent their time on the prowl at the BIO International Convention this week, searching for promising new drug candidates as money starts moving through the sector again.

A recent study published in PubMed explores the dissemination patterns of central nervous system Burkitt lymphoma, specifically from the fornix to the ventricles. This research may provide insights into the disease's progression and potential treatment strategies.