Don't Forget To
Rate This Article

Get the Latest Investment Ideas Delivered Straight to Your Inbox. Subscribe


Co. Uses RNA Editing as Treatment for Rare Disease
Research Report

Share on Stocktwits


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."

 AATD treatment represents an area of unmet need as no approved disease-modifying treatments are on the market.




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."

Want to be the first to know about interesting Biotechnology / Pharmaceuticals investment ideas? Sign up to receive the FREE Streetwise Reports' newsletter. Subscribe

1) Doresa Banning wrote this article for Streetwise Reports LLC and provides services to Streetwise Reports as an independent contractor. She or members of her household own securities of the following companies mentioned in the article: None. She or members of her household are paid by the following companies mentioned in this article: None.

2) The following companies mentioned in this article are billboard sponsors of Streetwise Reports: None. Click here for important disclosures about sponsor fees. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security. As of the date of this article, an affiliate of Streetwise Reports has a consulting relationship with: None. Please click here for more information.

3) Comments and opinions expressed are those of the specific experts and not of Streetwise Reports or its officers. The information provided above is for informational purposes only and is not a recommendation to buy or sell any security.

4) The article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility. By opening this page, each reader accepts and agrees to Streetwise Reports' terms of use and full legal  disclaimer. This article is not a solicitation for investment. Streetwise Reports does not render general or specific investment advice and the information on Streetwise Reports should not be considered a recommendation to buy or sell any security. Streetwise Reports does not endorse or recommend the business, products, services or securities of any company mentioned on Streetwise Reports.

5) From time to time, Streetwise Reports LLC and its directors, officers, employees or members of their families, as well as persons interviewed for articles and interviews on the site, may have a long or short position in securities mentioned. Directors, officers, employees or members of their immediate families are prohibited from making purchases and/or sales of those securities in the open market or otherwise from the time of the decision to publish an article until three business days after the publication of the article. The foregoing prohibition does not apply to articles that in substance only restate previously published company releases.

6) This article does not constitute medical advice. Officers, employees and contributors to Streetwise Reports are not licensed medical professionals. Readers should always contact their healthcare professionals for medical advice.

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.

The securities of the company discussed in this report may be unsuitable for investors depending on their specific investment objectives and financial position. Past performance is no guarantee of future results. This report is offered for informational purposes only, and does not constitute an offer or solicitation to buy or sell any securities discussed herein in any jurisdiction where such would be prohibited. This research report is not intended to provide tax advice or to be used to provide tax advice to any person. Electronic versions of H.C. Wainwright & Co., LLC research reports are made available to all clients simultaneously. No part of this report may be reproduced in any form without the expressed permission of H.C. Wainwright & Co., LLC. Additional information available upon request.

H.C. Wainwright & Co., LLC does not provide individually tailored investment advice in research reports. This research report is not intended to provide personal investment advice and it does not take into account the specific investment objectives, financial situation and the particular needs of any specific person. Investors should seek financial advice regarding the appropriateness of investing in financial instruments and implementing investment strategies discussed or recommended in this research report.

H.C. Wainwright & Co., LLC’s and its affiliates’ salespeople, traders, and other professionals may provide oral or written market commentary or trading strategies that reflect opinions that are contrary to the opinions expressed in this research report.

H.C. Wainwright & Co., LLC and its affiliates, officers, directors, and employees, excluding its analysts, will from time to time have long or short positions in, act as principal in, and buy or sell, the securities or derivatives (including options and warrants) thereof of covered companies referred to in this research report.

The information contained herein is based on sources which we believe to be reliable but is not guaranteed by us as being accurate and does not purport to be a complete statement or summary of the available data on the company, industry or security discussed in the report. All opinions and estimates included in this report constitute the analyst’s judgment as of the date of this report and are subject to change without notice.

Securities and other financial instruments discussed in this research report: may lose value; are not insured by the Federal Deposit Insurance Corporation; and are subject to investment risks, including possible loss of the principal amount invested.

Want to read more about Biotechnology / Pharmaceuticals investment ideas?
Get Our Streetwise Reports Newsletter Free and be the first to know!

A valid email address is required to subscribe