Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.
The cell and gene therapy sectors are growing exponentially, with new players emerging daily and much progress being made both in and out of the lab. CGTLive’s Around the Helix is your chance to catch up with the latest news in cell and gene therapies, including partnerships, pipeline updates, and more.
A phase 1/2 clinical trial (NCT05360238) has dosed its first patient with MB-106 (Mustang Bio), a chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed or refractory B-cell non-Hodgkin lymphomas and chronic lymphocytic leukemia.
The phase 1/2 MARVEL-1 clinical trial (NCT04040049) of Freeline Therapeutics’ FLT190, an investigational adeno-associated virus vector-based gene therapy intended to treat Fabry disease, has dosed the first patient in its second cohort.
The FDA has cleared Lyell Immunopharma’s investigational new drug application for LYL845, a tumor-infiltrating lymphocyte therapy to treat relapsed/refractory melanoma.
Allogene Therapeutics has initiated a phase 2 clinical trial (ALPHA2) for ALLO-501A, an investigational allogeneic anti-CD19 chimeric antigen T-cell (CAR-T) therapy, which will treat patients with relapsed/refractory large B-cell lymphoma.
Nanoscope Therapeutics’ MCO-010, an investigational ambient-light activatable multi-characteristic opsin optogenetic monotherapy, has received fast track designation from the FDA for the treatment of retinitis pigmentosaB via intravitreal injection.
The CardiAMP cell therapy showed clinical improvements in patients with heart failure, according to 2-year data from a phase 3 trial (NCT02438306).
IMA203, Immatics’ PRAME-targeted ACTenginecell therapy, has demonstrated clinical responses as a monotherapy in multiple forms of solid tumors, according to new data from a phase 1 dose-escalation trial (NCT03686124).
BrainStorm Cell Therapeutics’ NurOwn, an investigational autologous mesenchymal stem cell neurotrophic factor–secreting cell therapy product, demonstrated encouraging preliminary safety and efficacy data in an open-label phase 2 clinical trial (NCT03799718) in patients with primary or secondary progressive multiple sclerosis, according to a report published in Multiple Sclerosis Journal.
Atsena Therapeutics’ ATSN-101 (previously referred to as SAR439483), an investigational adeno-associated virus vector-based gene therapy intended to treat Leber Congenital Amaurosis caused by biallelic mutations in GUCY2D (LCA1), has demonstrated promising safety and efficacy in a phase 1/2 clinical trial (NCT03920007), according to data presented at the American Academy of Ophthalmology Annual Meeting which took place from September 30 – October 1, 2022, in Chicago, Illinois.
Lineage will also be receiving a patent from the European Patent Office for its retinal pigment epithelium cell manufacturing process.
The addition will support Ray Therapeutics' RTx-015, an investigational gene therapy for retinitis pigmentosa.
The collaboration will utilize ONI's imaging technology and will proceed with the aims of better understanding of CAR-T treatments and gathering information potentially useful for the development of future CAR-T therapies.
Cabaletta Bio intends to use IASO's clinically validated CD19 binder in its investigational CD-19 targeted CAR-T therapy CABA-201.
The patent assignment from CLS to Xenetic is related to a collaboration between the 2 companies and VolitionRx Limited focused on using Volition's Nu.Q technology and Xenetic's DNase-Armored CAR-T platform to develop new cell therapy treatments for solid tumors.
Ochre intends to use the funds to build off previous research with the goal of developing new investigational RNA-based therapies, which they intend to carry out preclinical studies with in their recently launched "Liver ICUs".