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Orchard Therapeutics: Updating The Investment Thesis

May 05, 2020 7:17 AM ETOrchard Therapeutics plc (ORTX)7 Comments


  • Today, we take a look at an intriguing biotech concern from across the pond called Orchard Therapeutics.
  • The company has multiple 'shots on goal' and several definable milestones on the horizon.
  • A full investment analysis follows in the paragraphs below.
  • I do much more than just articles at The Biotech Forum: Members get access to model portfolios, regular updates, a chat room, and more. Get started today »

Most doctors are prisoners of their education and shackled by their profession.

- Richard Diaz

A month ago we offered up the analysis below on this 'watch item' biotech stock from across the pond exclusively to Biotech Forum members. The stock is up over 40% since then but the concern is still intriguing. Today, we update our original thesis to account for all company-specific news and analyst ratings on this name since our original research was posted.

Company Overview:

Orchard Therapeutics (NASDAQ:ORTX) is a London, United Kingdom-based biopharmaceutical company that IPO’d in 2018. The company’s mission is to help patients with rare conditions by leveraging its understanding of gene therapy. The company’s approach is to take a patient’s own blood stem cells and insert into them a working copy of the missing or malfunctioning gene. The company’s approach circumvents the need for a bone marrow transplant by taking advantage of blood stem cells' intrinsic capacity to self-renew in a patient’s bone marrow and produce new blood cells of all types. The ultimate goal is to permanently correct genetic disorders through the use of a single treatment. The company’s pipeline is broken down into three overarching programs: neurometabolic disorders, primary immune deficiencies, and blood disorders. The company’s lead product candidates are OTL-200, OTL-101 and OTL-103. The company’s only commercial product is Strimvelis, which has been approved by the EMA but not by the FDA. Orchard Therapeutics has a market capitalization of just over $1 billion and trades for around $11 a share.


Source: Company Presentation


OTL-200 is an ex vivo autologous gene therapy in development to treat Metachromatic leukodystrophy. The drug uses a modified virus to insert an operational copy of the ARSA gene into a patient’s cells. OTL-200 has received rare pediatric disease designation from the FDA. MLD is a rare

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This article was written by

Bret Jensen profile picture
Finding tomorrow's big winners in the lucrative biotech sector

Finding tomorrow's big winners in the lucrative biotech sector, The Biotech Forum focuses on proprietary, breaking research on promising biotech and biopharma stocks with significant potential for outsized alpha. It is the fourth most subscribed to investment service offered through the Marketplace on SeekingAlpha.com. Our service offers a model-20 stock portfolio as well as the most active Live Chat on the Marketplace. This is where scores of seasoned biotech investors trade news and investment ideas back and forth throughout the trading day.

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Analyst’s Disclosure: I am/we are long ORTX. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Seeking Alpha's Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any investment is suitable for a particular investor. Any views or opinions expressed above may not reflect those of Seeking Alpha as a whole. Seeking Alpha is not a licensed securities dealer, broker or US investment adviser or investment bank. Our analysts are third party authors that include both professional investors and individual investors who may not be licensed or certified by any institute or regulatory body.

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