Conventional medicines treat the symptoms instead of the cause of diseases, meaning that patients must be treated throughout their lives. We are working to develop one-time treatments that target the cause of diseases.
Patients Are Counting on Us
Carlos, David and Vince launched UKATPA to educate and support patients, doctors and caregivers.
Genome Editing Resources
The resources below provide background information regarding cell and gene therapies, the Adapted from a naturally occurring bacterial immune system, CRISPR is an acronym for Clustered Regularly Interspaced Short Palindromic Repeats. One of the proteins in the CRISPR system is known as CRISPR-associated 9 protein or Cas9 protein, which acts as a pair of ‘molecular scissors’ to cleave DNA. Researchers have co-opted the bacterial CRISPR/Cas9 system to make specific changes in the DNA of humans, other animals and plants. CRISPR/Cas9 was first harnessed in 2012 as a genome editing tool in the lab. More recently, scientists have begun engineering and testing CRISPR systems to be very specific to a desired genetic target. technology and Intellia’s 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. work.
Intellia is developing therapies that edit patients’ somatic cells, which are the non-reproductive cells, to permanently treat their disease without impacting their progeny. Our therapies will not seek to edit patients’ germline or reproductive cells. (Changes to germline cells could be passed down to future generations.) Read Intellia’s full statement here.
Resources for the Transthyretin (ATTR) Amyloidosis Community
We are proud to work closely with the following organizations so that we can better understand the needs of patients and their families and caregivers. These organizations can also provide you additional information and access to Also known as ATTR amyloidosis. 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 ATTR amyloidosis, ATTRv or hATTR) or due to a natural form of this protein, without genetic mutation (wild-type ATTR amyloidosis, ATTRwt 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. research and support.
Resources for the Acute Myeloid Leukemia Community
Click the links below for more information about Also known as AML. Cancer of the blood and bone marrow that is rapidly fatal without immediate treatment, and is the most common type of acute leukemia in adults., Intellia’s second development program.
“Guide to Leukemia – Acute Myeloid – AML”
American Society of Clinical Oncology
“Adult Acute Myeloid Leukemia Treatment (PDQ®)–Patient Version”
National Cancer Institute
“Acute Myeloid Leukemia Disease Information”
Leukemia & Lymphoma Society
“What is AML?”
KNOW Also known as AML. Cancer of the blood and bone marrow that is rapidly fatal without immediate treatment, and is the most common type of acute leukemia in adults.
Resources for the Hereditary Angioedema Community
Click the links below for more information about Also known as HAE. Rare and potentially life-threatening genetic disease characterized by overproduction of bradykinin, which leads to recurring, severe and unpredictable swelling in various parts of the body., Intellia’s third development program.
“Your Guide to Understanding Genetic Conditions: Hereditary Angioedema”
NIH, U.S. National Library of Medicine – Genetics Home Reference
NIH, National Cancer for Advancing Translational Sciences – Genetic and Rare Disease Information Center
“What is HAE?”
US Hereditary Angioedema Association
“What is HAE?”