Wellcome Trust ISSF Cross-disciplinary grant awarded to Dr. Serpi and Dr. Pertusati

Dr. Serpi (PI)  and Dr. Pertusati in collaboration with Professor David Williams (Cardiff School of Dentistry) and Dr. Ian Fallis (Cardiff School of Chemistry) have been awarded the Wellcome Trust Cross Disciplinary Award with a project entitled ‘Novel antibiotic for the treatment of multidrug-resistant Gram-positive bacteria’.

This Grant will fund a cross-disciplinary research led to the optimization of a novel molecule active against antibiotic-resistant strains of Gram-positive bacteria.



NRN Translational fund awarded to Dr. Pertusati

Life Science Research Network translational fund has awarded 5000 GBP to Dr. Pertusati. The Translational Support Fund will allocate up to £5,000 to directly enhance the translational potential of existing drug discovery research projects. The fund will be used to study the ADME properties of our lead candidates in the treatment of lysosomal storage disease ( Niemann-Pick disease).

A Boost in Rare Disease Research Highlights Cardiff Chemists’ Platform Approach

On September 25, 2018, Cerecor Inc. (NASDAQ: CERC), a biopharmaceutical company focused on becoming a leader in development and commercialization of treatments for orphan disorders, announced its acquisition of Ichorion Therapeutics, Inc. Ichorion, a privately-held biopharmaceutical company based in New York City, focused on developing treatments for inherited metabolic disorders known as Inborn Errors of Metabolism (IEMs) and has supported a multi-year collaboration led by Dr. Fabrizio Pertusati of Cardiff University’s School of Pharmacy and Pharmaceutical Sciences. Dr. Pertusati and his research team have sought to expand the applicability of the ProTide platform, a chemical approach to nucleoside prodrugs that originated at Cardiff with Dr. Pertusati’s mentor and advisor, the late Dr. Chris McGuigan. The Cardiff chemists have already demonstrated the ProTide platform’s utility to combat infectious diseases and cancer, but the collaboration with Ichorion has provided a fresh look at therapeutic approaches for rare metabolic diseases the primarily effect children. These diseases typically have high infant mortality rates and no approved therapies.

“Our collaboration with Ichorion to research and develop new treatments for rare pediatric diseases is critically important to these under-served patient populations” said Dr. Pertusati. “The ProTide approach for IEM therapies is a logical extension of the foundational work that Chris, our lab and colleagues have done over the past 20 years. We’ve witnessed its success in virology and oncology, and now we believe we can leverage the platform to address rare metabolic diseases that primarily affect children with no treatment options.”

Cerecor continues to support advancing a pipeline of therapies for IEMs through its partnership with Cardiff University.

Exploring New Therapeutic Approaches to Treat Inborn Errors of Metabolism

Scientists in the School of Pharmacy and Pharmaceutical Sciences have teamed up with an industry collaborator to leverage their platform chemistry toward developing new therapies for rare pediatric diseases with no available treatment.

Inborn Errors of Metabolism (IEMs) are a group of inherited disorders caused by genetic defects that hinder production of vital metabolic intermediates, eventually leading to tissue myopathy and organ failure. In some cases, mortality rates are higher than 50% in early childhood.

Previous attempts at treating these diseases using a substrate replacement approach have generated proof-of-concept in in vitro models, demonstrating the ability to rescue depletion of key metabolites and salvage cell viability. However, the prospects of such approaches as pharmaceuticals have been complicated by a lack of optimal ‘drug-like’ properties.

Now, the medicinal chemistry group at Cardiff hopes to successfully apply knowledge gained transforming the treatment of anti-viral diseases to developing treatments for rare disorders in collaboration with Ichorion Therapeutics, a New York-based biopharmaceutical company focused on IEMs. Building upon the ProTide strategy originally conceptualized by Cardiff’s own Professor Chris McGuigan, the collaboration takes a fresh look at a prodrug approach balancing the capability to directly address a monogenic deficiency of the disease state with a small molecule possessing suitable chemical properties to become a medicine.

One such disease state is the mitochondrial DNA depletion syndrome (MDS) known as Deoxyguanosine Kinase (DGUOK) Deficiency. MDS patients have difficulty synthesizing mitochondrial DNA (mtDNA), which compromises the cell’s powerhouse. Cardiff and Ichorion have worked closely over the past year to optimize lead compounds shown to rescue mtDNA depletion in multiple models of DGUOK Deficiency. What they’ve learned is being applied to others MDSs and to IEMs more broadly.

Dr. Fabrizio Pertusati of Cardiff University’s School of Pharmacy and Pharmaceutical Sciences, who leads the chemistry efforts, added, ‘This work may represent an important step forward toward hope for patients with unmet medical needs. We’ve learned a lot applying our chemistry to anti-viral and anti-cancer drug development, and we’re excited to extend our scientific platform with the hope of helping under-served patient populations with rare metabolic diseases.’