CAMBRIDGE, Mass.--(BUSINESS WIRE)--RaNA Therapeutics, a leader in the discovery and development of a new class of medicines that target RNA, today presented preclinical data characterizing gene upregulation technology that selectively activates gene expression within the body’s own cells to treat spinal muscular atrophy (SMA). The data were presented on March 23, 2016 at the Keystone Symposium for Chromatin and Epigenetics in Whistler, British Columbia, Canada.
SMA is the leading genetic cause of infant mortality, affecting about 1 in 10,000 newborns. The primary symptom in SMA patients is motor function loss, likely resulting from early motor neuron loss. SMA is caused by mutations or deletions of SMN1, but SMN2 can compensate for SMN1 if its expression can be activated to produce functional protein. Using RaNA’s novel gene upregulation technology, increased expression of SMN protein has been achieved in preclinical models.
“These data are exciting, as they preclinically validate our scientific approach to selectively upregulating gene expression to increase protein levels in the body for therapeutic benefit,” said Ron Renaud, Chief Executive Officer of RaNA Therapeutics. “SMA is a devastating disease, and we are focused on rigorously exploring and advancing our oligonucleotide technology to develop treatment options that make a meaningful difference to patients.”
The poster presentation, titled A gene upregulation technology that selectively activates genes by targeting PRC2-associated lncRNA, presents findings that a novel long non-coding RNA (lncRNA) recruits a gene-repressive complex called PRC2 to SMN2, leading to its repression. By using a proprietary short oligonucleotide, PRC2 is sterically blocked from being recruited to SMN2. As a result, data show increased SMN mRNA and protein levels in SMA patient cells. This therapeutic approach is designed to target genes, rather than chromatin modifying complexes, with potential applications for many human genetic disorders.
In addition, RaNA’s oligonucleotide technology presented several key characteristics:
- Observation indicated sustained duration of action by oligonucleotides with broad bioavailability.
- Genome-wide analyses after oligonucleotide treatment showed highly specific activity.
- Single molecule RNA-FISH data demonstrated that the lncRNA functions in cis and that oligonucleotide treatment does not disrupt localization of this lncRNA, as further evidence supporting the specificity of RaNA’s approach.
On April 5, Balkrishen Bhat, Ph.D., RaNA’s Vice President of Chemistry, will also present as chairperson at HealthTech’s Oligonucleotide Therapeutics and Delivery Conference in Cambridge, Mass. In addition to presenting SMA and Friedreich’s ataxia data findings, Dr. Bhat will present in vitro and in vivo proof of concept of RaNA’s mRNA stabilization platform to increase the half-life and upregulation of mRNA and protein.
About RaNA Therapeutics
RaNA Therapeutics is a leading biotechnology company committed to the development of next generation RNA-targeted medicines that selectively upregulate gene expression to increase endogenous protein levels for therapeutic benefit. The company was founded by preeminent global leaders who pioneered the relationship between lncRNA and chromatic modifiers, as well as breakthrough oligonucleotide technologies. RaNA’s mission is to improve the lives of people suffering from serious life-altering diseases by creating precision medicines that can change the course of their condition. RaNA’s technology has broad therapeutic potential to treat a wide range of diseases, including rare genetic disorders. The company has current lead programs in spinal muscular atrophy and Friedreich’s ataxia. For more information about the company and its platforms, please visit www.ranarx.com.