In this interview, Professor Francesco Bifari guides us through the complex and fascinating world of Advanced Therapy Medicinal Products (ATMP). Together, we explore global development trends, the significant social impacts of regenerative medicine research, and the technological and regulatory challenges that characterize this rapidly evolving sector. Bifari also discusses the advancements of Hemera, with its REMaST® therapy, and the promising development prospects that could revolutionize the treatment of many neurological diseases.

1. What is the global development trend of Advanced Cell Therapies?

Advanced cell therapies fall under the classification of Advanced Therapy Medicinal Products (ATMP), which are human therapeutic products based on genes, tissues, or cells. ATMPs offer therapeutic opportunities for a significant number of severe diseases that have so far been considered orphan, meaning without any available therapy.

Currently, there are about 35 ATMPs on the market for various therapeutic indications, ranging from oncology to genetic diseases. However, there are more than 500 ATMP products in clinical trials. Over the past ten years, the development of ATMPs has primarily occurred in the United States and Europe, but growth is global. For instance, in China alone, around 400 ATMP products have been developed in the last 5 years.

2. What are the social impacts of research in regenerative medicine?

Research in regenerative medicine aims to find new therapies to regenerate tissues. It is already applied in many fields such as hematology, dermatology, and ophthalmology. Finding solutions to regenerate tissues that do not spontaneously regenerate, like nervous tissue, would represent a goal that could enable the treatment and cure of numerous neurological diseases.

3. How does Hemera position itself in this context?

Hemera has in its portfolio a product, REMaST®️, which has shown extraordinary capability in preclinical stages to regenerate nervous tissue, positioning itself at the forefront of the regenerative medicine sector.

4. What are the main challenges in developing advanced therapies?

There are mainly two: technological and regulatory.

Although the production methods of ATMPs are still relatively immature compared to other therapeutic products, such as antibodies, the technology for developing and producing ATMPs is rapidly evolving. Similarly, regulatory approaches for the clinical use of these innovative therapies are changing.

Many companies are now developing systems that allow for automated and closed-loop production (where the product does not come into contact with the operator), enabling standardized production.

To facilitate the regulatory process in Europe, the approval for the use of ATMPs is centralized and involves an evaluation of the product’s quality, safety, and efficacy by the Committee for Advanced Therapies (CAT) and subsequent approval by the Committee for Medicinal Products for Human Use (CHMP).

5. What are the development prospects of the REMaST therapy?

The therapeutic efficacy of REMaST®️ is based on multiple mechanisms of action, primarily the growth and maturation of neurons, which is complemented by a strong effect in promoting the growth of new vessels, remodeling fibrotic scars, and reducing inflammation.

We aim to test the efficacy of REMaST®️ in pathological contexts that could benefit from these mechanisms of action, such as neurological diseases where there has been a loss of nervous tissue.

6. What global standards guide the production process of advanced cell therapies?

Like all other drugs, cell therapies must ensure the safety and efficacy of each produced batch. Hemera is developing an automated and closed-loop production method for REMaST®️ , which ensures the same cellular product is obtained regardless of the production site.