sarepta therapeutics-2

Top 10 Series: Sarepta Therapeutics

Jason Myhre
4 min read

Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder that causes progressive muscle degeneration. The disorder is caused by a genetic mutation where the body is unable to produce a protein called dystrophin, and the effects of DMD are heartbreaking. One company in our Top 10* series is working to restore the lives of patients with DMD...


Company Name: Sarepta Therapeutics
Weight: 3.11% of Eventide Gilead (#6 of top 10 holdings)
Theme: Pediatric and Orphan Diseases

Duchenne Muscular Dystrophy (DMD) is a rare genetic disorder that causes progressive muscle degeneration. The disorder is caused by a genetic mutation where the body is unable to produce a protein called dystrophin. Dystrophin is the largest protein in the body and acts like a ‘shock absorber’ in the muscles. Without this shock absorber dampening the movements of the muscles, the PODs-1muscles tear, degrade, and die. The effects of DMD are heartbreaking: as the muscles degrade, patients lose the ability to walk, and eventually breath and pump blood. As a result, patients with DMD typically die in their 20s and 30s.

Sarepta Therapeutics is working to restore the lives of patients with DMD. Using gene therapy, Sarepta has found a way to deliver a healthy gene to their cells, enabling these patients to start producing a micro-form of dystrophin. This achievement was perhaps the biggest breakthrough in biotechnology in 2018. The promise of the therapy goes beyond halting the negative effects of the disease – to actually reversing its course and restoring muscle function once again. (Sarepta Therapeutics is based in Cambridge, MA.)

Don't want to wait for the next post? To learn about all ten companies in this series, check out the Eventide Gilead Portfolio Impact Highlights here:



*A "Top 10" series is a series of articles based on top 10 holdings of a fund by weight.

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Mutual funds involve risk including the possible loss of principal. Past performance does not guarantee future results. The fund can invest in smaller-sized companies which may experience higher failure rates than larger companies and normally have a lower trading volume than larger companies. The fund’s ethical values screening criteria could cause it to under perform similar funds that do not have such screening criteria. The fund can have risk associated with the biotechnology and pharmaceutical industry in which these companies may be heavily dependent on clinical trials with uncertain outcomes and decisions made by the U.S. Food and Drug Administration. Companies in the technology industries have different risks including but not limited to products becoming obsolete, and entrance of competing products. Companies in the Industrials sector carry various risks including, but not limited to, risk related to debt loads and intense competition. The fund can have risk related to option investing. There are special risks associated with investments in foreign companies including exposure to currency fluctuations, less efficient trading markets, political instability and differing auditing and legal standards. The fund can invest in private companies. Private investments include various risks including but not limited to lack of liquidity, capital commitment risk, and valuation risk. Private companies may not be financially profitable and have uncertain futures, subjecting them to additional risks.

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Posted June 10, 2019

Topic(s): Stories

Jason Myhre
Jason Myhre

Jason serves as Director of Marketing at Eventide. He is responsible for leading Eventide’s brand strategy, marketing, and corporate communications.