Around the Helix: Cell and Gene Therapy Company Updates – January 15, 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. First Patient With Multiple Myeloma Dosed in EsoBiotec's Trial for In Vivo CAR-T ESO-T01

The first patient has been dosed in an early phase 1 investigator-initiated clinical trial (NCT06691685) taking place in China for EsoBiotec's ESO-T01, an investigational BCMA-directed in vivo chimeric antigen receptor T-cell (CAR-T) therapy being evaluated for the treatment of relapsed/refractory multiple myeloma.

2. Atsena Therapeutics’ Makes Progress With X-Linked Retinoschisis Gene Therapy Trial

Atsena Therapeutics has launched Part B of its phase 1/2 LIGHTHOUSE clinical trial (NCT05878860) evaluating ATSN-201, an investigational adeno-associated virus (AAV) gene therapy, for the treatment of X-linked retinoschisis (XLRS) associated with mutations in the RS1 gene.

3. Ocugen’s Retinitis Pigmentosa Gene Therapy OCU400 Sustains or Improves Visual Function in 2-Year Follow-Up Data

Among 9 patients treated in the study who were evaluable at 2 years posttreatment, all 9 showed improved or preserved visual function in comparison to their untreated fellow eyes.

4. Verismo Therapeutics’ Trial for R/R B-NHL CAR-T SynKIR-310 Doses First Patient

The first patient has been dosed in Verismo Therapeutics’ phase 1 CELESTIAL-301 clinical trial (NCT06544265), which is evaluating SynKIR-310, a chimeric antigen receptor T-cell (CAR-T) therapy based on the company's killer immunoglobulin-like receptor (KIR)-CAR platform, in patients with relapsed/refractory (r/r) B-cell nonHodgkin lymphoma (B-NHL).

5. A2 Bio Garners $80 Million for Tmod CAR-T Programs

A2 Biotherapeutics has secured $80 million from investors in a Series C round of financing, with the proceeds to be put toward its development of CAR-T programs based on the company's Tmod platform technology. The Tmod approach is intended to allow CAR T-cell therapies to selectively kill tumor cells while sparing healthy tissue, by conferring the CAR T-cells with an “activator” that detects antigens typically found on tumor cells and a "blocker" that detects antigens typically found on healthy cells.

6. Angelini Ventures Coleads €20 Million Series A Round for RNA Therapeutic-Focused Company Neumirna Therapeutics

Neumirna Therapeutics, a company focused on the development of RNA-based treatments for neurology indications, has received €20 million in a Series A round of funding coled by investor Angelini Ventures. Neumirna's lead candidate is NMT.001, an antisense oligonucleotide therapy intended to provide a disease-modifying treatment option for drug-resistant epilepsy.

7. Orna Therapeutics and Vertex Team Up to Tackle Hemoglobinopathies With LNP-Delivered Gene Editing

Orna subsidiary ReNAgade Therapeutics and Vertex Pharmaceuticals have entered a strategic research collaboration agreement with the aim of developing next-generation gene editing approaches for the treatment of sickle cell disease and transfusion-dependent beta thalassemia. “Vertex is a leader in delivering next-generation approaches to treating hemoglobinopathies, and we are excited to collaborate with them to develop in vivo therapies that leverage our proprietary technologies to achieve unprecedented delivery to hematopoietic stem cells (HSCs),” Amit Munshi, MBA, the CEO of Orna, said in a statement. “[This] collaboration further validates our industry leading extrahepatic lipid nanoparticle (LNP) delivery chemistries and highlights the importance of delivery to enable the next wave of RNA medicines.”

8. Astraveus Produces CAR T-Cells With Its Microfluidic Benchtop System

According to the company, its end-to-end approach to manufacturing via its LakhesysBenchtop Cell Factory may enable a reduction of costs and a boost to throughput in comparison to traditional manufacturing processes. “Our approach has successfully miniaturized a hugely complex process, reducing the scale of the manufacturing system to the size of a book – less than one hundredth the size of existing technologies," Jérémie Laurent, PhD, the CEO of Astraveus, said in a statement. "With a production time of only 26 hours, we have also demonstrated that our bead-free microfluidic selection technology enables fast CAR-T cell manufacturing."