It was supposed to be an exciting time of next-gen science, where CRISPR gene editing was being used in the first series of human trials in the hope this tech could help cure a range of diseases.
But one of its proponents, CRISPR Therapeutics, has, alongside a growing number of biopharmas, admitted the spreading COVID-19 pandemic is starting to see it be “adversely affected.”
In a Securities and Exchange Commission filing Tuesday, the biotech said: “We are conducting a number of clinical trials for product candidates in the fields of severe hemoglobinopathies and immuno-oncology in geographies which are affected by the coronavirus pandemic.
“We believe that the coronavirus pandemic has had, and will likely continue to have, an impact on various aspects of our clinical trials. For example, with respect to our CTX001 clinical trials for severe hemoglobinopathies (specifically, transfusion-dependent beta thalassemia and severe sickle cell disease), since [intensive care unit] beds and related healthcare resources are anticipated to become significantly constrained in light of the coronavirus pandemic, no additional patients are currently scheduled to begin dosing in either study at this time.”
CTX001 is an investigational CRISPR gene-edited therapy for patients suffering from beta thalassemia and sickle cell disease in which a patient's hematopoietic stem cells are engineered to produce high levels of fetal hemoglobin in red blood cells. This is currently in a long-term follow-up trial after posting some encouraging, though early, results in several patients late last year.
Looking at its cancer trials, it also added that investigators will likely not want to expose cancer patients to COVID-19 “since the dosing of patients is conducted within an in-patient setting,” which could also hit it tests.
Its leading immuno-oncology cell therapy programs, CTX110 and CTX120, are using an allogenic approach to tumor targets. CTX110 targets CD19, an antigen expressed in various B-cell malignancies, while CTX120 targets BCMA, an antigen expressed in multiple myeloma.
Its third candidate, CTX130, is focused on solid tumors and targets CD70, an antigen expressed on both hematologic cancers, including certain lymphomas, as well as solid tumors including renal cell carcinoma.
CTX110 started an early safety trial last summer, and the biotech said in its financial report back in February that it was still enrolling patients to assess the safety and efficacy of CTX120. Meanwhile, CTX130 is yet to enter the clinic.
The biotech has already closed its offices and asked that most of its staffers work remotely. It said it has restricted on-site staff to only those personnel and contractors who “must perform essential activities that must be completed on-site and limited the number of staff in any given research and development laboratory.”
But this comes with its own issues: “Our increased reliance on personnel working from home may negatively impact productivity, or disrupt, delay, or otherwise adversely impact our business. In addition, this could increase our cyber security risk, create data accessibility concerns, and make us more susceptible to communication disruptions, any of which could adversely impact our business operations or delay necessary interactions with local and federal regulators, ethics committees, manufacturing sites, research or clinical trial sites and other important agencies and contractors.”
It also said that, given that its labs are not easily accessible and talking to regulators is getting tough, there could be a delay for the “timely completion” of preclinical activities, including IND-enabling studies as well as its ability to select future development candidates and initiation of additional clinical trials for other development programs.
Finances for the biotech may be hit, too. “The trading prices for our common shares and other biopharmaceutical companies have been highly volatile as a result of the coronavirus pandemic,” the company said. “As a result, we may face difficulties raising capital through sales of our common shares or such sales may be on unfavorable terms.”
The biotech has seen its shares yo-yo over the past month as COVID-19 ramped up—down from more than $53 a share at the start of March to less than $43 just two weeks later—and ended the month at around $42.
In short, CRISPR is braced for the worst but does not know how deeply this could cut into its trial and business. “We do not yet know the full extent of potential delays or impacts on our business, our clinical trials, our research programs, healthcare systems or the global economy. We will continue to monitor the situation closely.”