Sharetreck – Tracking Genomic Investments & clinical trials for HIV.

Introduction

In the world of genomics, progress often appears to move at a slow and steady pace, making it a prime opportunity for investors to deepen their understanding of the field and its potential. Today, we’re delving into CTX110, a promising gene therapy, its competitors, and the evolving landscape of B cell lymphoma treatment.

But before we dive into the exciting world of genomics, we kindly request that you subscribe to our channel if you haven’t already. Your support enables us to continue exploring groundbreaking developments in genomics.

CTX110 vs. BMS Breyanzi

As genomics enthusiasts, many of us have invested in CRISPR Therapeutics (CRSP) with the belief that gene editing and genomic medicines are the future of healthcare. Now, as we approach the monetization phase, it’s intriguing to witness the emergence of gene therapies in the pharmaceutical market. One such contender is CRISPR Therapeutics with its CTX110, poised to compete with BMS Breyanzi.

Currently, the target indication for CTX110, B cell lymphomas, is primarily treated with chemotherapy as the first line of defense. Only if chemotherapy fails and the patient cannot undergo a bone marrow transplant is BMS Breyanzi considered.

Breyanzi has demonstrated significant improvements in event-free survival (EFS), complete responses (CR), and progression-free survival (PFS) compared to standard therapy. What sets Breyanzi apart is its autologous nature—it uses the patient’s own T cells, genetically engineered for therapy. However, this approach requires lead time, cell collection centers, and a specialized treatment infrastructure.

Moreover, Breyanzi cannot be used as a first-line treatment due to concerns about cytokine syndrome and neurologic toxicities.

CTX110’s Superiority

CTX110, on the other hand, is an allogenic therapy, offering distinct advantages. Let’s explore why CTX110 is a superior offering.

TCR Knockout: CTX110 leverages TCR knockout technology to mitigate cytokine release syndrome (CRS) and neurotoxicity, common side effects of CAR-T cell therapy. TCR-deficient CAR-T cells have the potential to reduce alloreactivity, lowering the risk of graft-versus-host disease (GVHD) and off-target toxicities.

Challenges and Considerations: While TCR-deficient CAR-T cells may decrease CRS, they might also be less effective in recognizing and destroying target cells, as TCRs play a crucial role in the immune response. Safety concerns, optimal design, and individualized approaches must be considered in the development and clinical application of TCR-deficient CAR-T cells.

MHC-1 Expression: To ensure CAR-T cell persistence, MHC-1 expression is suppressed. However, this strategy carries the risk of natural killer (NK) cells targeting CAR-T cells lacking MHC-1 expression.

Competitors in the Market

Aside from CTX110 and Breyanzi, the gene therapy market boasts other well-established players, such as Yescarta and Kymriah. Both Yescarta and Kymriah are autologous therapies.

In terms of pricing, Breyanzi is listed at $410,000, Yescarta at approximately $424,000, and Kymriah at $475,000.

In 2022, BMS generated $122 million from Breyanzi, while Gilead earned $337 million from Yescarta, reflecting a 6% growth from the previous year. Kymriah logged in an impressive $536 million in revenue.

CTX-110: The Future Revenue Driver

Considering the substantial revenue generated by autologous therapies, one can only imagine the potential for an off-the-shelf allogenic therapy like CTX-110. Notably, CTX-110 is fully owned by CRISPR Therapeutics.

CRISPR’s Challenge and Innovation

CRISPR Therapeutics faces the challenge of avoiding cytokine release syndrome while maintaining CAR-T cell persistence. While TCR knockout helps reduce CRS, the suppression of MHC-1 expression may attract NK cells. It’s possible that CRISPR is fine-tuning MHC-1 downregulation, a topic that may be explored further in upcoming clinical trial data.

Promising Developments

As per CRISPR’s Q2 earnings report, they are actively enrolling and dosing patients in a Phase 2 clinical trial of CTX110, which targets CD19+ B-cell malignancies. Encouraging preliminary data has granted CTX110 RMAT designation by the FDA, potentially leading to a landmark approval.

Conclusion

In conclusion, the world of genomics and gene therapies is on the cusp of groundbreaking advancements. CTX110, with its allogenic approach, presents an exciting opportunity in the treatment of B cell lymphomas. As investors, we can find comfort in CRISPR Therapeutics’ promising developments and the potential it holds for the future of medicine.