Janneke de WAL
Director Clinical Development, Amsterdam Molecular Therapeutics
Dr Janneke de Wal is Director of Clinical Development a.i. at Amsterdam Molecular Therapeutics (AMT) B.V..
AMT, founded in 1998 and based in Amsterdam, the Netherlands is a leader in the development of gene therapies. People at AMT aspire to use their know-how and expertise to significantly improve the lives of patients with serious, debilitating disorders.
Janneke de Wal has almost 25 years pharmaceutical industry experience, in medical information, project management, early stage and advanced commercial development and clinical development. She has worked in many therapeutic areas, during recent years focusing on orphan disease. Prior to joining AMT Dr de Wal worked for Genzyme in the (orphan disease) area of lysosomal storage disorders.
Dr. de Wal received an M.D. from the Medical School of the Rijks Universiteit Leiden.
GlyberaŽ- working together to bring safe and efficacious gene therapy to LPLD patients (Not available)
A continuing problem in the management of the majority of inherited disorders is the unavailability of effective disease-modifying therapy rather than measures that merely alleviate symptoms. Many of these conditions are rare, and development of conventional causal therapies is impossible. Yet, all such disorders together affect millions of people and represent a great need for improvement, and for the application of new therapy paradigms, like gene therapy.
We are developing innovative therapies making use of functioning genes 'packaged' in specific viral vectors to target relevant organs and systems. The development of Glybera in lipoprotein lipase deficiency (LPLD) is the first AMT application of this principle; we observed that a one time intramuscular administration of Glybera leads to long term reduction of pancreatitis episodes, the most severe, lethal manifestation of this disease.
Our dream is to ultimately provide such a hope for cure to many people affected by genetic disorders, using this new platform of therapy.