Cystic Fibrosis clinical advisory board
Client: Proteostasis Therapeutics
Consultant: Professor Jane Davies, National Heart and Lung Institute
Expertise: Pharmaceuticals, Drug development, Cystic Fibrosis(CF), Clinical trials
Our consultants shared their expertise in Cystic fibrosis (CF), drug development and clinical trials to help Proteostasis Therapeutics assess the combination of compounds aimed at treatments to improve lung function.
Proteostasis is a drug discovery company addressing diseases caused by defects in protein folding, trafficking and clearance for orphan and neurodegenerative diseases, including Cystic Fibrosis (CF).
Proteostasis needed expert advice on the selection process of the company’s leading compounds aimed at improving lung function that are due for clinical development.
The compounds double the activity of the drug combination ivacaftor/lumacaftor in the gold-standard test using Human Bronchial Epithelial cells (HBE cell assay) which is a drug combination that has been shown to significantly improve lung function in sufferers with the most common CF mutation, F508del/F508del, and it was hoped that the compounds selected for further development can build on this.
The work at hand required stratetic planning over a period of time and Professor Jane Davies, along with five other world leading experts, was appointed to Proteostasis Therapeutics’ Cystic Fibrosis clinical advisory board, which was given the task to serve as a strategic resource for the upcoming selection and study of the compounds.
Following the appointment of experts on the board and the insights gained from their involvement, Meenu Chhabra, President and Chief Executive Officer of Proteostasis Therapeutics said:
The knowledge and experience that our inaugural clinical advisory board members bring to Proteostasis will be invaluable not only in the selection process of which of our proprietary PTI CFTR compounds should proceed to clinical development, but also in providing guidance on the design and conduct of the future clinical trials of selected drug candidates.
President and CEO, Proteostasis Therapeutics
Related case studies
Our consultants shared their expertise in clinical medicine to help IMS Health to assess the adoption, governance and regulatory landscape of electronic medical records (EMR) at different levels of healthcare systems around the world. read more
Genotype and resistance testing for new direct-acting antiviral (DAA) drugs designed to treat Hepatitis C virus (HCV). read more
The Imperial College London Diabetes Centre was established in Abu Dhabi, in 2006, by Mubadala and experts from Imperial to address the demand for diabetes care in the UAE. With continued support from Imperial, the centre now operates three branches across Abu Dhabi and Al Ain and public health initiatives. read more