Recombinant human Laminin-111 for treatment of Duchenne Muscular Dystrophy

Dr Hodges
Prothelia Inc, USA

Sunday 6th June 2010

TACT agreed with Prothelia that this is an early stage proposal. The group recommended that more preclinical data should be generated and these experiments are specifically listed in the report to the applicant. Evaluating the activity of recombinant laminin 111 to increase alpha7-integrin expression levels will be an important experiment, as well as the assessment of more functional outcomes in mdx mice and possibly also GRMD dogs.

Provided experts then agree that results of these experiments support progress to a clinical phase, substantial involvement of clinicians and trial experts will be necessary to develop clinical protocols. The applicant clearly recognizes that there is a large volume of manufacturing process development, preclinical pharmacology, and toxicology work to be accomplished before the launch of human clinical trials. TACT agrees, but emphasizes the additional key preclinical questions to be addressed before initiating planned GLP toxicology and subsequently clinical studies.

12 Apr 2017