Wave Life Sciences to Present Preclinical In Vivo and In Vitro Data for SNP3-Targeting Huntington’s Disease Program at CHDI Foundation’s 15th Annual HD Therapeutics Conference
Wave’s HD pipeline includes its Phase 1b/2a investigational oligonucleotides targeting SNP1 (WVE-120101) and SNP2 (WVE-120102), as well as its SNP3-targeting oligonucleotides. All of these oligonucleotides are designed to be allele-selective and preferentially lower mHTT protein while keeping the level of healthy or wild-type HTT (wtHTT) protein relatively intact. The wtHTT protein is important for neuronal function, and there is increasing evidence that it may be neuroprotective in an adult brain. Additionally, a dominant gain of function in mHTT protein and a concurrent loss of function of wtHTT protein may be important components of the pathophysiology of HD.
Details of Data Presentation
- Poster: Stereopure oligonucleotides for the selective silencing of mutant huntingtin
Wednesday, February 26, 2020
1:00-4:00 p.m. PST
About Wave Life Sciences’ Clinical Research in Huntington’s Disease
Wave is developing WVE-120101 and WVE-120102, which are investigational stereopure antisense oligonucleotides designed to selectively target the mutant huntingtin (mHTT) mRNA transcript of SNP
WVE-120101 and WVE-120102 are currently being investigated in early manifest HD patients in the Phase 1b/2a PRECISION-HD1 and PRECISION-HD2 studies, respectively. In
In addition to WVE-120101 and WVE-120102, Wave expects to initiate clinical development of a SNP3-targeting candidate in the second half of 2020. Between its SNP1, SNP2 and SNP3 programs, Wave has the potential to address up to 80% of the HD patient population. Wave’s allele-selective approach may also enable the company to address the pre-manifest, or asymptomatic, HD patient population in the future.
About Huntington’s Disease
Huntington’s disease (HD) is a debilitating and ultimately fatal autosomal dominant neurological disorder, characterized by cognitive decline, psychiatric illness and chorea. HD causes nerve cells in the brain to deteriorate over time, affecting thinking ability, emotions and movement. HD is caused by an expanded cytosine-adenine-guanine (CAG) triplet repeat in the huntingtin (HTT) gene that results in production of mutant HTT (mHTT) protein. Accumulation of mHTT protein causes progressive loss of neurons in the brain. HD may also be caused by a reduction in wild-type, or healthy, HTT (wtHTT) protein that occurs concurrently with production of mHTT protein. wtHTT protein is critical for neuronal function and suppression may have detrimental long-term consequences. Approximately 30,000 people in
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended, including, without limitation, the importance of wtHTT protein preservation in treating HD; Wave’s intention to explore higher doses in its PRECISION-HD1 and PRECISION-HD2 studies and the expected timing for future data readouts; the expected timing for the initiation of clinical development for Wave’s SNP3-targeting candidate; the potential addressable HD patient population covered by Wave’s three HD programs; and that Wave’s allele-selective approach may enable it to address the pre-manifest, or asymptomatic, HD patient population in the future. The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, the risks and uncertainties described in the section entitled “Risk Factors” in Wave’s most recent Annual Report on Form 10-K filed with the
Source: Wave Life Sciences