Intellia Therapeutics Inc. (NASDAQ:NTLA) has announced results of the quarter ended September 30, 2020, in which its collaboration revenue was $22.2 million. The revenue was mainly a result of $15.3 million realized following the transfer of control of the development licence for Factor VIII haemophilia A target related to the extension of Regeneron partnership.
Intellia advancing therapy for treating ATTR patients
The company’s CEO and President John Leonard said that the company recently received regulatory authorization to commence its Phase 1 NTLA-2001 study putting the company on track to dose first patients by the end of this year. This is a huge milestone in improving the quality of life of ATTR patients through the provision of a curative treatment. It marks Intellia’s transition into a clinical-stage company. Advancing NTLA-2001 is vital in genome editing because this will be the first clinical study of a systemically delivered CRISPR/Cas9-based therapy.
Leonard confirmed that the company is also progressing NTLA-2002 and NTLA-5001 development in parallel for treatment of HAE and AML respectively to an IND or equivalent submission in 2021. The company continues to develop novel capabilities across its platform based on the CRISPR/Cas9 technology in generating therapeutic candidates.
UK MHRA grants Intellia approval for first-in-human NTLA-2001 clinical study
Recently the company announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) to commence first-in-human NTLA-2001 clinical trial. NTLA-2001 is the company’s experimental therapy currently under development for treating clinical manifestations of ATTR. The therapy applies Antellia’s in vivo liver gene knockout tech, which allows for potential lifelong transthyretin protein reduction following a single. The study will evaluate adult patients with hereditary ATTR with polyneuropathy.
Antellia is advancing the AML program with NTLA-5001 which is a T Cell receptor cell therapy candidate that targets Wilms’ Tumor 1 antigen for treating AML. The company wants to develop the therapy as a broadly applicable AML treatment irrespective of cancer mutational subtypes. Antellia is advancing Investigational New Drug applications efforts, and it’s on track to submit an INDA for NTLA-5001 in 1H 2021.
