The 62° AFI National Symposium opens with an initial session devoted to the evolution of advanced therapies for safer, more effective and accessible drugs for patients.
The search for new therapies is unstoppable, and now all biotech companies are investing in the development of advanced therapies. Now rare diseases, sometimes referred to as orphan diseases, are the subject of numerous studies, and an increasing number of diseases are moving toward possible cures.
Among these it is worth mentioning the development of neural stem cell-based therapies to treat some neurological diseases, while gene and personalized therapy is increasingly at the forefront, targeting the treatment of diseases of liver metabolism.
Dr. Maria Luisa Nolli told us about all this in her interview with MakingLife, as she is personally involved in the pharmaceutical industry, which has always been aimed at promoting the development of these new technologies that have changed and will continue to change the face of medicine.
The development of advanced therapies requires complex steps, a lot of time and dedication, starting with the research groups engaged in the development of new vectors particularly for gene therapy and CarT therapy; only after lengthy in vitro and in vivo trials can one speak of clinical trials involving several steps to market approval of the drug.
Biotech companies involved in the development of ATMPs (Advanced Therapy Medicinal Products) face many challenges, foremost among them economic challenges given the fact that these treatments are often personalized, thus targeting a single patient, or at most a niche of patients with a rare disease. The best approach for large pharmaceutical companies to develop these drugs is to outsource the research and development part to external entities, often universities, thus lowering costs. Only if the results of subsequent trial phases are promising do companies invest more funds on the drug in development. This business model is proving successful within the pharmaceutical biotech arena.
With a mention of the PNRR, Dr. Nolli reminds how important it is not to miss the opportunity to present new and innovative projects that can bring funds to university research centers and biotech companies, as well as the fact that it is a great opportunity for networking between laboratories and companies from different backgrounds, all devoted to the advancement of advanced therapies.