Expanded Access Policy
Intellia is committed to solving the complex challenges of making CRISPR/Cas9-based therapies a reality for patients suffering with severe and life-threatening genetic diseases, including diseases such as Also known as ATTR. A rare genetic disease caused by accumulation of misfolded transthyretin (TTR) protein, which affects the nerves, heart, kidneys and eyes. Patients can develop amyloidosis by inheriting the faulty TTR gene from a parent (hereditary or hATTR) or due to a natural form of this protein, without genetic mutation (wild-type or wtATTR). NTLA-2001, Intellia’s first investigational therapy, is being evaluated in a Phase 1 clinical trial as a treatment for people who have hereditary ATTR with polyneuropathy. (Also known as ATTR. A rare genetic disease caused by accumulation of misfolded transthyretin (TTR) protein, which affects the nerves, heart, kidneys and eyes. Patients can develop amyloidosis by inheriting the faulty TTR gene from a parent (hereditary or hATTR) or due to a natural form of this protein, without genetic mutation (wild-type or wtATTR). NTLA-2001, Intellia’s first investigational therapy, is being evaluated in a Phase 1 clinical trial as a treatment for people who have hereditary ATTR with polyneuropathy.). In these efforts, Intellia is guided by ethical, scientific and legal principles, which require well-designed and controlled clinical trials to evaluate the safety and efficacy of our potential In a research or clinical setting, investigational means that the drug has not been approved or authorized for use in patients outside of a clinical trial by any authority that regulates new treatments, such as the U.S. Food and Drug Administration (FDA) or United Kingdom Medicines and Healthcare products Regulatory Agency (MHRA). Also called gene editing. Genome editing collectively refers to a set of technologies, including CRISPR/Cas9, that can be used to cut and modify DNA. Genome editing uses systems to make the DNA change inside the cell. These cells can be edited in the body (in vivo) or outside the body (ex vivo) from a patient or donor. therapies. Click here to visit the National Institutes of Health (NIH) website to learn more about clinical trials and how they work.
Under certain circumstances, a person suffering a serious or life-threatening disease may ask to use an experimental treatment outside a A test of a new medical treatment or procedure in human volunteers with the purpose of evaluating a medical, surgical or behavioral intervention. Clinical trials typically follow preclinical (non-human) studies and are the primary way that clinical researchers and regulatory agencies evaluate whether a new treatment is safe and effective in people., before its safety and efficacy have been fully evaluated, and before the regulatory authorities have approved it. Generally, this is an option only for patients who have exhausted all available medical options and do not qualify for the ongoing clinical trials. The U.S. Food and Drug Administration (FDA) explains such “expanded access” as follows:
Expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Intellia appreciates the intent of expanded access programs but, at this time, we can best advance the development of these potential promising products by enrolling patients in clinical trials. Our clinical trials will be designed, conducted and monitored to ensure that the safety and effectiveness of our experimental therapies are properly evaluated before they are made available generally. Should you have any questions, please reach out to firstname.lastname@example.org or speak with your physician. Intellia expects to acknowledge receipt of requests sent to this email address within five business days.
Intellia reserves the right to revise this policy at any time.