PTC Therapeutics Selects New SMA Drug Development Candidate

19 August 2013

PTC Therapeutics has announced that it has selected and will continue to develop a candidate drug that has the potential to treat SMA.

Triggering a $10 million payment from Roche to PTC, this marks the first major development in the multi-million dollar collaboration between PTC, Roche, and theSMA Foundation, initiated in late 2011 (for more information on this deal click here).

PTC is trying to identify and develop drugs that are able to target post-transcriptional control mechanisms. These are regulatory events taking place in our bodies once DNA (genes) has been copied into messenger RNA (mRNA), but before the mRNA is used to produce protein (click here for a refresher on how DNA is used as a template to produce protein).

Post-transcriptional control mechanisms affect the expression of our genes and include processes such as splicing (i.e. the removal of stretches of mRNA that do not encode information about how to build a protein) and capping, which serves to stabilise the mRNA such that it survives for longer in the cell before being naturally broken down.

For SMA, PTC aims to identify and develop drugs able to target these control mechanisms in order increase the production of the survival motor neuron (SMN) protein, the levels of which are reduced in SMA patients.

Representatives of PTC, Roche, and the SMA Foundation are all excited by the news, as it represents a step in the right direction for SMA therapy development.


Further Information

PTC Therapeutics Press Release