Cell therapy weekly: CDMO partners with patient advocacy group
This week: The UK Medicine and Healthcare products Regulatory Agency (MHRA; London, UK) authorized a Clinical Trial Application for a CRISPR-based gene editing therapy, a US$8 million grant was awarded to support the development of CAR-T therapy for relapsed/refractory AL Amyloidosis, and a partnership was formed between a CDMO and a patient advocacy group to advance FOXG1 gene therapy.
The news highlights:
- UK MHRA authorizes gene editing therapy indicated for a rare inherited disease to go to trial
- CAR-T therapy for relapsed/refractory AL Amyloidosis gets CLIN2 funding
- Collaboration with a patient advocacy group to advance FOXG1 gene therapy
UK MHRA authorizes gene editing therapy indicated for a rare inherited disease to go to trial
The UK MHRA has authorized Intellia Therapeutics’ (MA, USA) Clinical Trial Application for NTLA-3001, an in vivo CRISPR/Cas9-based gene editing treatment designed to treat a rare inherited disease called alpha-1 antitrypsin deficiency (AATD)-associated lung disease.
NTLA-3001 aims to correct the deficiency by inserting a functional SERPINA1 gene, potentially restoring normal levels of the AAT protein with a single treatment and providing an alternative to current treatment methods, which include long-term AAT replacement therapy or lung transplants.
The Phase I/II trial has been approved to commence in late 2024 to test the safety and effectiveness of NTLA-3001 and will include a dose-escalation phase involving up to 30 patients.
“NTLA-3001 is a groundbreaking in vivo CRISPR-based gene insertion candidate designed to durably produce functional AAT protein at normal levels after a one-time treatment. We are excited to receive regulatory authorization to begin this important first-in-human study of NTLA-3001 for people living with AATD,” said John Leonard, Intellia President and CEO. “In addition, this study serves to validate our modular gene insertion platform, which we plan to leverage to address numerous diseases caused by a missing or defective protein.”
CAR-T therapy for relapsed/refractory AL Amyloidosis gets CLIN2 funding
The California Institute for Regenerative Medicine (CA, USA) has awarded a CLIN2 grant of US$8 million to Nexcella (CA, USA), a cell therapy division of Immix Biopharma (CA, USA), to support the clinical development of the company’s investigational CAR-T therapy for relapsed/refractory AL Amyloidosis.
The CAR-T therapy, NXC-201, targets B-cell maturation antigen and is intended to be a one-time therapy. Nexcella will use the funding to further the development of the therapeutic for the Phase Ib/II dose expansion trial, NEXICART-2. The trial is expected to enroll patients with adequate cardiac function who have not had prior B-cell maturation antigen-targeted therapy to evaluate the safety and efficacy of NXC-201.
“AL amyloidosis is an unmet medical need and current approved therapies for this rare disease are mostly not well tolerated and have not led to sustained remissions,” stated Abla Creasey, VP of Therapeutics Development at California Institute for Regenerative Medicine.
Collaboration with a patient advocacy group to advance FOXG1 gene therapy
Charles River Laboratories International (MA, USA) has joined forces with a patient advocacy group, the FOXG1 Research Foundation (FRF; NY, USA), to propel gene therapy development for FOXG1 syndrome, a severe neurological disorder. The partnership aims to streamline FRF’s path to clinical trials by combining FRF’s model platforms for patient data and Charles River’s cell and gene therapy expertise and CDMO facilities.
Charles River will produce materials for FRF’s Phase I/II AAV vector-based gene therapy clinical trials, including High Quality plasmid starting materials and good manufacturing practice AAV9 viral vectors, at its centers of excellence (CoE), Alderley Park CoE (Chesire, UK) and Rockville CoE (MD, USA).
“Charles River is proud to work with the FOXG1 Research Foundation to advance its gene therapy through clinical trials,” commented Kerstin Dolph, Corporate Senior Vice President, Global Manufacturing, Charles River. “The FOXG1 patient population has an incredible unmet need, and we are looking forward to lending our expertise to FRF as they continue to trailblaze a path toward providing rare disease treatments.”
