Cell therapy weekly: TGA grants green light to in vivo CAR-T cell therapy
This week: A study detailed findings of a potential target for T-cell therapy in acute myeloid leukemia (AML), US$170 million was raised in Series B funding to advance research in retinal gene therapy. Plus, the Australian Therapeutic Goods Administration (TGA; Canberra, Australia) has given the green light for an in vivo chimeric antigen receptor (CAR) therapy for B-cell malignancies to be assessed in clinical trials.
The news highlights:
- Data demonstrates a potential target for acute myeloid leukemia T-cell therapy
- US$170 million in Series B funding raised to propel eye gene therapy research
- TGA gives the green light for an in vivo CAR therapy for B-cell malignancies to be assessed in clinical trials
Data demonstrates a potential target for acute myeloid leukemia T-cell therapy
Immunotherapy company CERo Therapeutics Holdings (CA, USA) presented data on its lead compound, CER-1236, at the Global Cell & Gene Therapy Summit 2024 (8-10 July 2024; Boston, Massachusetts). The presentation demonstrated the product’s potential to treat AML by specifically targeting TIM-4-L.
TIM-4-L is as a novel target for engineered T-cell therapy due to its high frequency of expression in AML cell lines. Additionally, the safety assessment demonstrated no adverse effects on healthy tissue and CERo aims to advance CER-1236 into clinical trials.
“In the meantime, with all [Investigational New Drug]-enabling work complete, we are in close communication with the [FDA] as we prepare for the possibility of entering the clinic with CER-1236 for AML in the short term,” shared Brian Atwood, Chairman and CEO of CERo.
US$170 million in Series B funding raised to propel eye gene therapy research
Beacon Therapeutics (FL, USA), an ophthalmic gene therapy company, has raised US$170 million in series B funding to advance the development of AGTC-501, a gene therapy program for X-linked retinitis pigmentosa and to support its dry age-related macular generation program for Phase I/II data collection.
Investors include Forbion (Naarden, Netherlands), TCGX (CA, USA), Syncona Limited (London, UK), Oxford Science Enterprises, the University of Oxford (both Oxford, UK) and Advent Life Sciences (London, UK).
Beacon is currently assessing AGTC-501 in a photoreceptor damage due to X-linked retinitis pigmentosa and mitigate rod and cone loss through AGTC-501’s ability to express an entire retinitis pigmentosa GTPase regulator protein.
“Forbion is pleased to support Beacon Therapeutics at this critical juncture in the development of its pipeline of pioneering ophthalmic gene therapies. Beacon’s deep expertise and renowned management bench give us confidence in the plan to build a leading retinal gene therapy company,” expressed Dmitrij Hristodorov, General Partner at Forbion and newly appointed member of the Board of Directors of Beacon.
TGA gives the green light for an in vivo CAR therapy for B-cell malignancies to be assessed in clinical trial
Interius BioTherapeutics (PA, USA) has received Clinical Trial Notification (CTN) clearance from the TGA for its lead in vivo CAR candidate, INT2104, which is indicated for B-cell malignancies. This clearance follows approval from the Human Research Ethics Committee (HREC) in Australia.
Following this milestone, Interius will start a first-in-human trial by the end of 2024.
“The approval allows us to enroll patients in our first clinical study and recognizes the potential of our novel in vivo targeted gene therapy candidate, INT2104, to address an unmet medical need for patients with B cell malignancies,” expressed Phil Johnson, CEO and President of Interius. “We look forward to continuing to work closely with the TGA and other regulators in the future to bring this innovative therapy to patients as quickly as possible.”
