CRISPR-Based Therapies Revolutionize the Treatment of Sickle Cell Disease, Underwhelm Investors

The field of gene editing has taken a leap forward with the recent approvals of two CRISPR-based drugs, Casgevy and Lyfgenia, for the treatment of sickle cell disease (SCD). These groundbreaking therapies offer potential for treating this previously incurable disease, generating excitement and hope for patients and the science around gene editing.

The Money

Pricing: Casgevy (CRISPR) $2.2 million, no black box. Lyfengia (Bluebird Bio) $3.1 million, black box warning for hematologic malignancy. These are some of the most expensive drugs available.

Vertex and CRISPR have significantly more financial resources than Bluebird. Vertex expects to reach nearly $10 billion in annual revenue by the end of 2023, while Bluebird is facing financial difficulties. According to their latest financial statements, Bluebird only has enough money to operate until halfway through 2024. The company was counting on selling a priority review pediatric voucher from the FDA to Novartis for $103 million to improve its financial situation, but the FDA ultimately denied their request.

On the day of the announcement, Bluebird closed down 40% while CRISPR Therapeutics closed down 6.7%. Bluebird Bio’s stock is down about 98% from its peak almost ten years ago. As of the end of September, Bluebird had $165 million in cash on hand, while it was burning through more than $70 million a quarter.

Despite its financial challenges, Bluebird still has one key advantage: they have been conducting an extensive pilot program that essentially serves as a dress rehearsal for the launch of their SCD. Bluebird’s Zynteglo gene therapy, designed for a similar blood disorder called beta thalassemia, is nearly identical to their sickle cell treatment. For the past year, Bluebird has been carefully rolling out Zynteglo to a smaller population, which has allowed them to identify and address any potential logistical challenges that might arise with the sickle cell gene therapy launch. This pilot program effectively serves as a “red carpet” rollout for the sickle cell treatment, ensuring a smoother and more successful launch.


A Historic First: Casgevy Blazes the Trail

On December 8th, 2023, the U.S. Food and Drug Administration (FDA) approved Casgevy, developed by Vertex Pharmaceuticals and CRISPR Therapeutics, as the first-ever CRISPR-based drug in the US. This historic decision paves the way for a new era in medicine, where gene editing holds the promise of curing countless diseases. Casgevy’s Mechanism of Action: Editing the Root Cause of SCD.

SCD is a debilitating and life-threatening inherited blood disorder affecting millions worldwide. The disease is caused by a single genetic mutation that leads to the production of abnormal red blood cells, which become rigid and sickle-shaped. These deformed cells can block blood flow, causing severe pain, anemia, organ damage, and shortened life expectancy.

Casgevy works by correcting the mutation. Using CRISPR/Cas9 technology, the therapy edits the mutated gene in the patient’s bone marrow stem cells. These edited cells are then reintroduced into the body, where they begin producing healthy red blood cells, free from the SCD mutation.

Clinical Trials Demonstrate Remarkable Efficacy

Clinical trials of Casgevy have shown remarkable efficacy. The therapy significantly reduced the frequency of painful episodes, also known as vaso-occlusive crises (VOCs), by as much as 90%. Additionally, Casgevy improved patients’ overall health and quality of life, reducing their need for pain medication and blood transfusions.

Lyfgenia Joins the Fight: A Second Option for SCD Patients

Just hours after Casgevy’s approval, the FDA also approved Bluebird Bio’s Lyfgenia (lovo-cel), another CRISPR-based therapy for SCD. Lyfgenia works similarly to Casgevy, utilizing CRISPR/Cas9 technology to edit the genetic mutation responsible for the disease.

Promising Results from Lyfgenia’s Clinical Trials

Like Casgevy, Lyfgenia demonstrated impressive efficacy in clinical trials. The therapy significantly reduced the frequency of VOCs and improved patients’ quality of life, offering another valuable option for those living with SCD.

Two Therapies, One Goal: A Brighter Future for SCD Patients

The approval of both Casgevy and Lyfgenia marks a significant milestone in the fight against SCD. These therapies offer patients hope for a future free from the debilitating symptoms of the disease. Additionally, the competition between companies is likely to drive down costs and accelerate the development of even more effective treatments in the future.

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CRISPR-Based Therapies Revolutionize the Treatment of Sickle Cell Disease, Underwhelm Investors was last modified: December 9th, 2023 by Simons Chase