Around the Helix: Cell and Gene Therapy Company Updates – June 4, 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. Cilta-Cel Benefit Consistent Across High-Risk Subgroups of Multiple Myeloma

Ciltacabtagene autoleucel (cilta-cel; Carvykti) improved progression-free survival (PFS) compared with standard of care (SOC) across all subgroups of patients with relapsed/refractory multiple myeloma, including in those with extramedullary disease (EMD) and regardless of prior lines of therapy, according to a subgroup analyses from the phase 3 CARTITUDE-4 trial (NCT04181827) presented at the 2025 ASCO Annual Meeting.

2. Iovance Biotherapeutics’ Lifileucel Produces Durable Responses in 5-Year Analysis of Advanced Melanoma Data

Iovance Biotherapeutics’ lifileucel (marketed as Amtagvi), an FDA-approved autologous tumor infiltrating lymphocyte (TIL) therapy, has produced durable responses in a 5-year analysis of data from patients with advanced melanoma treated the phase 2 C-144-01 clinical trial (NCT02360579).

3. Immix Biopharma’s CAR-T NXC-201 Effects Deep Hematologic Responses in 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 generated deep hematologic responses in patients with relapsed/refractory (r/r) light chain (AL) amyloidosis who were treated in the phase 1b/2 NEXICART-2 clinical trial (NCT06097832), which is taking place in the United States.

4. Satri-cel Makes Strides in Gastric and Gastroesophageal Junction Cancer

New results from the phase 2 pivotal trial of satricabtagene autoleucel (satri-cel), a Claudin18.2 (CLDN18.2)-targeting autologous CAR T-cell therapy in development by CARsgen underscore the growing potential of engineered cell therapies in solid tumors, showing positive data for patients with advanced gastric or gastroesophageal junction cancer.

5. Carlyle and SK Capital Bring bluebird bio into the Fold

Carlyle and SK Capital have acquired bluebird bio and have stated their intention to continue providing bluebird's commercially approved gene therapy products for sickle cell disease, β-thalassemia, and cerebral adrenoleukodystrophy. "We believe providing bluebird the necessary funding along with the new leadership team will help bluebird realize its full potential,” Joe Bress, a Carlyle partner and the global cohead of healthcare, said in a statement.

6. STEMCELL Technologies Scoops Up Cellular Highways

STEMCELL Technologies has acquired the biotech company Cellular Highways, which focuses on cell sorting technology that can be utilized for various applications, including cell and gene therapy development. Notably, the acquisition provides STEMCELL with Cellular Highways' Highway 1 instrument, which provides high-speed cell sorting via the company's proprietary Vortex-Actuated Cell Sorting (VACS) technology.

7. Beam Therapeutics Snags Orphan Drug Designation for BEAM-101

Beam has acquired orphan drug designation from the FDA for its investigational base-edited autologous hematopoietic stem cell therapy BEAM-101, which is intended to treat sickle cell disease. The therapy is currently being evaluated in the phase 1/2 BEACON clinical trial (NCT05456880).

8. Sarepta Garners Platform Technology Designation for Limb-Girdle Muscular Dystrophy Gene Therapy Vector

Sarepta Therapeutics has received a platform technology designation from the FDA for bidridistrogene xeboparvovec (also known as SRP-9003)'s rAAVrh74 viral vector. SRP-9003 is an investigational adeno-associated virus (AAV) vector-based gene therapy product in development for limb-girdle muscular dystrophy Type 2E (LGMD2E/R4, also known as beta sarcoglycanopathy). “This is one of the first programs to receive platform technology designation and an important recognition by FDA of the reproducibility and adaptability of this technology across multiple therapeutic programs,” Louise Rodino-Klapac, PhD, the chief scientific officer and head of research & development at Sarepta, said in a statement.