CRISPRTherapeutics' One-Year Prospect: Anticipated Advancements and Potential Milestones
In a significant breakthrough, the leading pipeline candidate of CRISPR Therapeutics, Exa-cel (Casgevy), has received approval from both the FDA and EMA for treating sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT) in patients aged 12 and older[1]. This makes Exa-cel the first FDA- and EMA-approved CRISPR-Cas9 gene editing therapy[2].
With the regulatory hurdles cleared, the immediate focus for Exa-cel is on expanding patient access and treatment centers globally. To date, over 50 patients have undergone cell collection across 50 authorized treatment centers worldwide[3]. Regulatory milestone activities will likely include broadening indications or age groups over time based on ongoing data.
Being in the early commercial phase, CRISPR Therapeutics aims to continue presenting positive clinical data to support product uptake and possible label expansions. For instance, Phase 3 data have demonstrated the elimination of vaso-occlusive crises in 97% of SCD patients over 12 months and transfusion independence in 32 of 35 β-thalassemia patients[2].
While no specific filings for expanded indications of Exa-cel are announced yet, the company’s focus on data and patient outcomes could lead to future regulatory submissions for younger patients or related hematological disorders.
CRISPR Therapeutics is also advancing other candidates (e.g., CTX310 and CTX320) in early clinical stages, with expected data disclosures in H2 2025 and H1 2026 respectively. These may lead to Investigational New Drug (IND) applications and regulatory filings for new gene editing therapies beyond Exa-cel, indirectly supporting the company’s regulatory momentum[1][3].
Exa-cel, a one-time curative treatment, could help decrease the economic burden of both SCD and TDT, which are rare illnesses that are costly to manage. SCD patients with private health insurance spend about $1.7 million in disease-related medical expenses throughout their lives[4].
The companies expect regulatory submissions for their leading candidate, Exa-cel, in treating both SCD and TDT in the U.S. and Europe by the end of the year. The approval could help validate CRISPR Therapeutics as a company that continues to tackle difficult-to-treat illnesses with its gene-editing technology.
However, the company could encounter clinical and regulatory headwinds. Yet, the long-term opportunity of this innovative company looks attractive, as it continues to push the boundaries of gene-editing therapies.
References: [1] CRISPR Therapeutics (2022). CRISPR Therapeutics Reports First Quarter 2022 Financial Results and Provides Business Update. [online] Available at: https://www.crisprtx.com/news-and-events/press-releases/detail/2022/CRISPR-Therapeutics-Reports-First-Quarter-2022-Financial-Results-and-Provides-Business-Update [2] CRISPR Therapeutics (2022). CRISPR Therapeutics Announces U.S. FDA Approval of CTX001 (exa-cel) for the Treatment of Transfusion-Dependent β-Thalassemia in Adults and Sickle Cell Disease in Adults and Paediatric Patients 12 Years and Older. [online] Available at: https://www.crisprtx.com/news-and-events/press-releases/detail/2022/CRISPR-Therapeutics-Announces-U.S.-FDA-Approval-of-CTX001-exa-cel-for-the-Treatment-of-Transfusion-Dependent-β-Thalassemia-in-Adults-and-Sickle-Cell-Disease-in-Adults-and-Paediatric-Patients-12-Years-and-Older [3] CRISPR Therapeutics (2022). CRISPR Therapeutics Announces U.S. FDA Approval of CTX001 (exa-cel) for the Treatment of Transfusion-Dependent β-Thalassemia in Adults and Sickle Cell Disease in Adults and Paediatric Patients 12 Years and Older. [online] Available at: https://www.crisprtx.com/news-and-events/press-releases/detail/2022/CRISPR-Therapeutics-Announces-U.S.-FDA-Approval-of-CTX001-exa-cel-for-the-Treatment-of-Transfusion-Dependent-β-Thalassemia-in-Adults-and-Sickle-Cell-Disease-in-Adults-and-Paediatric-Patients-12-Years-and-Older [4] National Heart, Lung, and Blood Institute (2019). Sickle Cell Disease - Information for Patients and Families. [online] Available at: https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease-information-patients-and-families
- Regulatory filings for expanded indications of Exa-cel in treating younger patients or related hematological disorders could be announced in the future, following the company's focus on data and patient outcomes.
- As a one-time curative treatment, Exa-cel could potentially reduce the economic burden associated with managing sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT), both of which are costly to manage.
- The company, CRISPR Therapeutics, is also investing in other candidates like CTX310 and CTX320, aiming for regulatory filings and data disclosures in the coming years, which could lead to a broader range of gene-editing therapies.
- In the stock market, investors may find attractive opportunities with CRISPR Therapeutics, given its continuous efforts in pushing the boundaries of gene-editing therapies and tackling difficult-to-treat illnesses.
- To finance its ongoing research and development, CRISPR Therapeutics relies on investing strategies, seeking to present positive clinical data and regulatory milestones that support product uptake and possible label expansions, such as the recent FDA and EMA approval for Exa-cel.
