Translating research ideas into prescription medicines is a long journey that can take between 12 to 15 years before becoming a reality. This enormous investment of time and effort is required to tackle all the steps in between – from choosing a therapeutic molecule, testing its safety and efficiency to finalising its formulation and mode of delivery to the patients. A crucial step in this process includes a regulatory approval from the corresponding authority such as the European Medicines Agency (EMEA) and other national bodies. Regulators need to be convinced that a new drug meets the standards for efficacy, safety and quality prior to its authorization and commercialization.

The onus is upon those developing a new drug to demonstrate four things to the regulatory authority for its approval: 1) the drug can be produced consistently, which means that every batch has to be identical,  2) the drug is efficient and has a desired therapeutic effect for the disease or condition in question, 3) the drug is safe and, 4) the drug has a favourable benefit-risk profile, meaning that the potential therapeutic effects of the treatment outweigh the potential side effects.

Within the New Deal Project, we aim to treat intestinal bowel disease (IBD) using molecules called siRNAs which target genes involved in intestinal inflammation. Our partner Boyd Consultants is responsible for guiding the consortium with the steps required for the regulatory approval of the project outcome. “Getting through a regulatory approval process involves regular interactions and meetings with regulators requiring significant scientific advice. We bring this key expertise into the New Deal group. In addition, we also help design the clinical studies that will demonstrate that the drug outcome of the New Deal project is safe and effective for patients”, explains Prof. Alan Boyd, director and founder of Boyd Consultants.

Prof. Boyd has been involved in bringing more than 15 medicines to the market through different regulatory bodies, including antibiotics, drugs to treat respiratory illness, cancer and heart failure. Furthermore, he is a pioneer in developing gene-based therapies. “When I started to work on gene-based therapies, there were no regulations for these treatments. People thought I was crazy because nobody has ever done it before!”. Today, the regulations for these novel therapies are clear. According to Prof. Boyd, “when designing and then manufacturing a gene-based therapy, researchers have to test three important aspects of the product: First, potency – which is if the product produces the desired effect, identity – to see if the drug has the right structure or if is it changing and finally, purity – making sure that the final material is free of impurities including viruses or bacteria”.

“Developing gene-based therapies is a bit more straightforward than a chemical medicine such as an antibiotic, and this often surprises people”, states Dr Boyd. When it comes to toxicology assessments where the potentially harmful effects of a drug are studied, gene-based therapies have a clear advantage over their chemical counterparts. “Gene-therapies typically do not have adverse effects in biological processes or functions such as fertility, reproduction or cancer, so the toxicology work is a lot shorter. Moreover, human clinical trials for chemical drugs usually start in healthy volunteers, while gene-based therapies can be tested directly in patients to assess their safety and efficacy as the first step rather than healthy volunteers”, explains Prof. Boyd.

However, within the New Deal Project, our partners are developing a lipid nanoparticle to carry the siRNA through the gastrointestinal tract of the patients so that the therapy is delivered at the intended organ.  “Of course, this affects the manufacturing of the product and its toxicology. We will also have to test the combination of siRNA inside the nanoparticle because that is what we hope to be giving to patients”, explains Prof. Boyd.

The regulatory challenges in bringing a novel therapy from bench to the bedside are not unique to the New Deal project – other nanomedicine initiatives such as B-smart, CUPIDO and Smart4Fabry currently face similar hurdles in developing their nanotechnology-based therapy for neurodegenerative diseases, cardiovascular disease and Fabry LSD disease, respectively. In collaboration with these sister projects, the New Deal consortium has organised a workshop to address the common “Translational issues in nanomedicine” to be conducted online on the 15th of October 2020. Attendees will have a chance to listen to Prof. Boyd´s take on the importance of a clinical development plan and regulatory milestones for nanomedicine projects. “We will be able to share what we have done in terms of developing the product, identifying its structure, the initial toxicology results, the planned clinical program and, of course, we will talk about some of the problems we have had along the way”, says Prof. Boyd.

The workshop is open to all registered participants and further details will be posted soon on our NewDeal website. To stay abreast of this and other developments in this project, don´t forget to subscribe to our newsletter here – https://bit.ly/33VkXDc