Co. Uses RNA Editing as Treatment for Rare Disease
Source: Andrew Fein
October 4, 2022 (Investorideas.com Newswire) The firm plans to advance its alpha-1 antitrypsin deficiency program into the clinic next year, noted an H.C. Wainwright & Co. report.
WAVE Life Sciences Ltd. (WVE:NASDAQ) is preparing to commence in 2023 a Phase 1/2 clinical trial of WVE-006, its treatment candidate for the rare genetic disease, alpha-1 antitrypsin deficiency (AATD), reported H.C. Wainwright & Co. analyst Andrew Fein in a September 29, 2022 research note. The next step is submitting a clinical trial application.
The genetic medicines company's AATD program involves using its ADAR RNA editing platform to target the single-base mutation in the SERPINA1 Z allele, an approach Fein described as "early but compelling."
WVE-006 is designed to correct the mutant SERPINA1 transcript and push AATD patients from a homozygous to heterozygous phenotype. This, in theory, would address the lung and liver problems associated with AATD and reduce patients' disease burden.
WAVE discussed both platform and program in its September 28 event called "Towards the Clinic: Spotlight on RNA Editing for AATD," which Fein attended.
One of Fein's takeaways, noted in his report, is that "mechanistically, the ability to increase the circulating levels of functional alpha-1 antitrypsin (M-AAT), while reducing liver aggregation arising from mutated protein, could provide a viable route of treatment" for AATD.
Preclinical data, Fein relayed, show that nearly 60% editing was achieved of the Z allele messenger RNA in the liver of transgenic human ADAR mice.
This "is indicative of levels nearing correction to heterozygote, while maintaining physiological regulation of M-AAT," wrote Fein.
However, on the downside, Fein pointed out, off-target effects were seen, a phenomenon that will need to be addressed.
Paucity of Disease-Modifying Treatments
As for the potential market size and current treatment of this rare hereditary disease, Fein noted an estimated 100,000 Americans may have Pi homozygous AATD, but only 10% are diagnosed. AATD treatment represents an area of unmet need as no approved disease-modifying treatments are on the market.
Fein explained that AATD causes pulmonary endothelial apoptosis resulting in emphysema. AATD patients typically present with respiratory symptoms such as wheezing, dyspnea, cough, and decreased exercise tolerance. The only approved treatment for pulmonary disease in severe AATD patients is augmentation therapy.
"Therefore, any [AATD] disease-modifying therapy needs to consider both the liver and the lungs, which has remained challenging so far," Fein wrote. WAVE's AATD program "could bring a differentiated approach to the developmental landscape."
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Disclosures For H.C.Wainwright & Co., WAVE Life Sciences Ltd, September 29, 2022
H.C. Wainwright & Co, LLC (the "Firm") is a member of FINRA and SIPC and a registered U.S. Broker-Dealer.
I, Andrew S. Fein, Ananda Ghosh, PhD, Andres Y. Maldonado, PhD and Matthew Caufield , certify that 1) all of the views expressed in this report accurately reflect my personal views about any and all subject securities or issuers discussed; and 2) no part of my compensation was, is, or will be directly or indirectly related to the specific recommendation or views expressed in this research report; and 3) neither myself nor any members of my household is an officer, director or advisory board member of these companies.
None of the research analysts or the research analyst's household has a financial interest in the securities of WAVE Life Sciences Ltd. (including, without limitation, any option, right, warrant, future, long or short position). As of August 31, 2022 neither the Firm nor its affiliates beneficially own 1% or more of any class of common equity securities of WAVE Life Sciences Ltd..
Neither the research analyst nor the Firm knows or has reason to know of any other material conflict of interest at the time of publication of this research report.
The research analyst principally responsible for preparation of the report does not receive compensation that is based upon any specific investment banking services or transaction but is compensated based on factors including total revenue and profitability of the Firm, a substantial portion of which is derived from investment banking services.
The firm or its affiliates received compensation from WAVE Life Sciences Ltd. for non-investment banking services in the previous 12 months. The Firm or its affiliates did not receive compensation from WAVE Life Sciences Ltd. for investment banking services within twelve months before, but will seek compensation from the companies mentioned in this report for investment banking services within three months following publication of the research report. The Firm does not make a market in WAVE Life Sciences Ltd. as of the date of this research report.
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