Gene Therapy Shows Promise But Still Has A Ways to Go

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Gene therapy has the potential to be a game-changing therapeutics to correct genetic “errors” that can cause rare diseases and illnesses. By replacing a mutated gene causing the disease with a healthy copy, doctors can treat certain patients without the use of surgery or pharmaceuticals.

In a sense, gene therapy combines therapeutics and engineering to help solve genetic issues within an individual. This makes gene therapy a major potential breakthrough in being able to treat several diseases, including inherited disorders, certain cancers & viral infections.

However, gene therapy remains a very risky endeavor. The U.S. Food and Drug Administration (FDA) has recently been taking a stand against gene therapy companies and holding them to higher standards. While gene therapy shows promise in being able to treat genetic diseases, the safety concerns, and limited treatment scope, serve as short-term hurdles for the industry.

FDA Hits Back at Gene Therapy Companies 

Emerging therapeutics are exciting and important for society’s ability to fight disease and ailments. However, newer treatment methods need to be held to the fire to ensure their safety and efficacy. Based on the recent actions by the FDA, this is exactly the game plan with gene therapy companies.

In August 2020, the FDA was expected to approve a highly-anticipated gene therapy treatment for hemophilia A called Roctavian, which is developed by BioMarin Pharmaceutical (NASDAQ: BMRN). The FDA denied the approval and subsequently asked BioMarin to gather more data on Roctavian.

Right around the same time, Audentes Therapeutics was the subject of more bad news for gene therapy companies, after a third clinical trial participant died after receiving the experimental therapy for a rare neuromuscular disease.

Just months after the BioMarin and Audentes setbacks, the gene therapy industry was hit with two more setbacks. UniQure (NASDAQ: QURE) is investigating whether a study participant’s liver cancer was the result of its experimental gene therapy treatment for hemophilia B. Sarepta Therapeutics (NASDAQ: SRPT), widely considered to be among the leaders in the industry, faces scrutiny after its treatment for Duchenne muscular dystrophy missed one of the main goals in clinical trials.

Looking back at 2020, the FDA stepped up its scrutiny of gene and cell therapy companies. The consistent feedback from the FDA has been to provide greater transparency into production processes. In other words, the FDA is sending a message to gene therapy companies that they need to up their standards and quickly.

Bluebird Splits Gene Therapy and Cancer Businesses

 Bluebird Bio (NASDAQ: BLUE), a component of the Investors Prism Emerging Gene Therapy Index, rose to prominence in recent years, thanks in part to its emerging gene therapy pipeline. However, after a few high-profile setbacks, the company recently announced it will be splitting the company into two segments: gene therapy and cancer.

“You don’t build an oncology company by hiring people who are experts in severe genetic disease, nor do you do vice versa,” Leschly told WSJ. “A lot of this comes down to … priorities and focus.”

In recent years, Bluebird has struggled to convert promising clinical data into commercialized therapies. Despite impressive data for its experimental sickle cell and multiple myeloma gene therapies, the company has been unable to translate it into a commercial product.

Additional companies developing gene therapies featured within the PRISM Emerging Gene Therapy Index include Orgenesis, Inc. (NASDAQ: ORGS), Celyad Oncology (NASDAQ: CYAD), Avrobio, Inc. (NASDAQ: AVRO), OncoSec Medical, Inc. (NASDAQ: ONCS), and more.

Overall, gene therapy continues to be a potential breakthrough to treat a wide range of inherited and rare diseases. However, given its past safety lapses, the industry continues to face scrutiny from the FDA and other health regulators to ensure a viable treatment. While COVID-19 is the primary focus right now, gene therapy has seen a renaissance, as companies look to get ahead of other emerging health threats in the future.

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Gene Therapy Shows Promise But Still Has A Ways to Go

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