Around the Helix: Cell and Gene Therapy Company Updates – February 12, 2025

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.

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1. Immix Biopharma’s CAR-T NXC-201 Snags FDA RMAT Designation for Light Chain Amyloidosis

Immix Biopharma and its subsidiary Nexcella's NXC-201, an investigational autologous BCMA-directed chimeric antigen receptor T-cell (CAR-T) therapy, has received regenerative medicine advanced therapy (RMAT) designation from the FDA for the treatment of relapsed/refractory (r/r) light chain (AL) amyloidosis.

2. Creative Medical Technology’s Cell Therapy AlloStem Reduces Insulin Dependency in Late-Stage Type 2 Diabetes

Creative Medical Technology’s AlloStem (also known as CELZ-201), an investigational universal and proprietary allogenic cell line, has demonstrated the ability to reduce insulin dependency among patients with type 2 diabetes (T2D) treated in a pilot study.

3. EGenesis Deems Second Transplant of Genetically Modified Porcine Kidney EGEN-2784 Successful

EGenesis has successfully transplanted EGEN-2784, a porcine kidney that features a number of genetic modifications intended to prevent rejection in human patients, into a second patient, with the intention of treating end-stage kidney disease.

4. NKGen’s Autologous NK Cell Therapy Troculeucel Fast Tracked for Alzheimer Disease

NKGen Biotech's troculeucel (also known as SNK01), an investigational autologous natural killer (NK) cell therapy, has been granted fast track designation by the FDA for the treatment of moderate Alzheimer disease.

5. MaxCyte and TG Therapeutics Team Up to Bring Allogeneic CAR-T to Autoimmune Disease

MaxCyte and TG Therapeutics have signed a strategic platform license allowing TG to utilize MaxCyte's Flow Electroporation technology and ExPERT platform in the development of azercabtagene zapreleucel (azer-cel), an investigational allogeneic CAR-T therapy, for autoimmune disease indications. Notably, TG Therapeutics licensed azer-cel from its owner Precision BioSciences, for development in nononcology indications.

6. NecstGen to Provide Manufacturing Services for Galapagos in Europe

Contract development and manufacturing organization (CDMO) NecstGen has entered a collaboration agreement with Galapagos NV through which it will provide manufacturing services in Europe for Galapagos' CAR-T candidates. "By combining our state-of-the-art infrastructure with Galapagos’ decentralized cell therapy approach, we aim to provide capacity and support for decentralised manufacturing and make transformative therapies more accessible to patients who need them," Paul Bilars, the CEO of NecstGen, said in a statement.

7. Cure Rare Disease Secures CIRM Grant to Support Spinocerebellar Ataxia ASO Research

The California Institute for Regenerative Medicine (CIRM) has provided a grant worth $5.69 million to Cure Rare Disease for the purpose of supporting the latter's development of an antisense oligonucleotide (ASO) therapy for the treatment of spinocerebellar ataxia type 3 (SCA3). The therapy is currently in preclinical development, but Cure Rare Disease intends to use the grant to support investigational new drug (IND)-enabling activities and submission of an IND.

8. Akribion Therapeutics Emerges From Stealth and Announces Focus on New RNA-Guided Technology

Akribion Therapeutics has emerged from stealth and announced its completion of a round seed financing led by CARMA FUND and RV Invest that garnered €8 million. Akribion is focused on the development of an RNA-guided technology that is intended to effect programmable cell depletion for therapeutic purposes. "Akribion is unlocking a whole new class of therapeutic possibilities with our approach," Lukas Linnig, MBA, the cofounder and coCEO of Akribion, said in a statement. "Our proprietary G-dase E nucleases allow very specific depletion of certain subsets of cells based on the presence of a predetermined RNA sequence. While we are initially focused on advancing groundbreaking precision oncology therapies, we believe that this approach to programmable cell depletion could have much wider application."