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We will need substantial additional financing to conduct our planned pivotal clinical trial for vispa-cel and to implement our operating plans.
If we fail to obtain additional financing, we will be unable to complete the development and commercialization of our vispa-cel and/or CB-011 product candidates.
Risks Relating to our Business, Government Regulation, Technology, and Industry Our CAR-T cell therapy product candidates are in clinical development and it will be many years before we commercialize a product candidate, if ever.
Changes at the FDA may hinder the agency s ability to hire and retain key leadership and other personnel, slow the time necessary for new product candidates to be reviewed and/or approved, or otherwise prevent these agencies from performing normal business functions on which our operations rely, which would adversely affect our business.
The regulatory landscape that will govern our product candidates is uncertain; regulations relating to more established gene therapy and cell therapy products are still developing, and changes in regulatory requirements could result in delays or discontinuation of development of our product candidates or unexpected costs in obtaining regulatory approval.
In the event that we are unable to continue to fund the clinical development of one or both of our product candidates, and/or if one or both of our clinical-stage product candidates is not clinically successful, does not receive regulatory approval, and/or is not commercially competitive, we currently do not have a research pipeline from which to generate new product candidates.
Our allogeneic CAR-T cell therapy product candidates will be regulated as biologics, and therefore may be subject to uncertainty regarding nonpatent regulatory exclusivity or maintaining regulatory approval.
We may not remain in compliance with the continued listing requirements of Nasdaq, and, if we are not able to remain in compliance, our common stock will be subject to delisting.
Our genome-editing platform is based on our novel chRDNA ( C RISPR h ybrid R NA- DNA , or chRDNA, pronounced chardonnay ) genome-editing technology, which enables more precise genome editing of allogeneic cell therapies.
Our allogeneic CAR-T cell therapy product candidates in clinical development are directed at CD19 and B cell maturation antigen ( BCMA ), established cell surface targets against which autologous CAR-T cell therapeutics have already demonstrated clinical proof of concept.
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Form 10-K Summary 115 i Tabl e of Contents Risk Factors Summary Our business is subject to a number of risks of which you should be aware before making a decision to invest in our common stock.
We will need substantial additional financing to develop our product candidates and implement our operating plans.
If we fail to obtain additional financing, we may be delayed or unable to complete the development and commercialization of our product candidates.
Risks Relating to our Business, Government Regulation, Technology, and Industry We are early in our product development efforts and it will be many years before we commercialize a product candidate, if ever.
Our business is highly dependent on the success of our product candidates, which will require significant additional human clinical trials before we can seek regulatory approval and potentially commercialize our product candidates.
If we are unable to advance our clinical trials and obtain regulatory approval for, and successfully commercialize, our product candidates for the treatment of patients in approved indications, or if we are substantially delayed in doing so, our business will be materially harmed.
There is substantial uncertainty regarding the new Administration s initiatives and how these might impact the FDA, its implementation of laws, regulations, policies, and guidance, and its personnel.
Similar initiatives may also be directed toward other government agencies.
These initiatives could prevent, limit, or delay development and regulatory approval of our product candidates, which would adversely affect our business.
We face significant competition from other biotechnology and pharmaceutical companies, which may result in other companies developing or commercializing products before, or more successfully than, we do, thus ii Tabl e of Contents rendering our product candidates non-competitive or reducing the size of the market for our product candidates.