Around the Helix: Cell and Gene Therapy Company Updates – December 4, 2024

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. FDA Announces Probe Into bluebird's Elivaldogene Autotemcel for Hematologic Malignancies

The FDA has announced it will be investigating a number of reports it has received on bluebird bio's approved autologous hematopoietic stem cell (HSC)-based gene therapy elivaldogene autotemcel (eli-cel; marketed as Skysona), related to incidence of hematologic malignancies. In its announcement, the agency noted that some of these cases have included life-threatening cases of myelodysplastic syndrome and acute myeloid leukemia in patients with early, active cerebral adrenoleukodystrophy.

2. REGENXBIO Doses First Patient in Phase 3 Trial for DMD Gene Therapy RGX-202

REGENXBIO has dosed the first patient in the pivotal phase 3 portion of its phase 1/2/3 AFFINITY DUCHENNE clinical trial (NCT05693142), which is evaluating RGX-202, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of Duchenne muscular dystrophy (DMD).

3. Genethon and Hansa Biopharma Launch Trial for Gene Therapy and Antibody Cleaving Enzyme Therapy Combination Treatment in Crigler-Najjar Syndrome

Genethon and Hansa Biopharma have initiated a phase 2 clinical trial (GNT-018-IDES; NCT06518005) evaluating Hansa’s immunoglobulin G antibody cleaving enzyme therapy imlifidase (marketed in Europe under the name Idefirix) as a pretreatment for Genethon’s GNT-0003, an investigational AAV serotype 8 vector-based gene therapy intended to treat severe Crigler–Najjar syndrome.

4. SC291 CAR T-Cell Therapy Fast Tracked for Relapsed/Refractory SLE

Sana Biotechnology’s investigational chimeric antigen receptor (CAR) T-cell therapy, known as SC291, has been granted fast track designation by the FDA for the treatment of relapsed or refractory systemic lupus erythematosus (SLE), including both lupus nephritis and extrarenal lupus.

5. Roche to Bring Poseida Into the Fold

Poseida Therapeutics, which is developing allogeneic T stem cell memory cell (T_SCM)-rich CAR-T therapies, has entered into an agreement to be acquired by Roche Holdings, Inc. "We have worked closely with Roche through our collaboration focused on hematologic malignancies, and we are excited to join Roche to work as colleagues together across our pipeline and future programs," Kristin Yarema, PhD, the president and chief executive officer of Poseida, said in a statement. "Roche's global capabilities in late-stage development and commercialization will enable patients worldwide to benefit from the transformative potential of allo CAR-T."

6. Sana Biotechnology's CAR-T Fast Tracked in Lupus

The FDA has granted fast track designation to Sana Biotechnology's SC291, an investigational allogeneic CAR-T therapy that is hypoimmune (HIP)-modified and targets CD19, for the treatment of relapsed/refractory systemic lupus erythematosus. SC291 is currently being assessed in the phase 1 GLEAM clinical trial (NCT06294236) for the treatment of lupus nephritis, extrarenal lupus, and antineutrophil cytoplasmic antibody-associated vasculitis.

7. Exegenesis Bio's Fabry Disease Gene Therapy EXG110 Garners Orphan Drug Designation

Exegenesis Bio's EXG110, an investigational gene therapy intended to treat Fabry disease, has received orphan drug designation from the FDA. EXG110 is currently being evaluated in a clinical trial in China, in which the first patient has been dosed. A planned clinical trial for patients in the United States is expected to launch in 2025.

8. Amarna Therapeutics Enlists the Aid of NorthX Biologics for Gene Therapy Manufacturing

Under a new partnership, NorthX Biologics will provide manufacturing services for first in human clinical trials for Amarna Therapeutics' Nimvec AM510, an investigational gene therapy intended to treat immune-mediated diseases. ”We are excited to collaborate with the Amarna team, allowing us to leverage NorthX Biologics’ expertise in viral vector manufacturing, to support the development of this pioneering gene therapy platform," Janet Hoogstraate, PhD, the CEO of NorthX Biologics, said in a statement. "Together, we aim to accelerate the journey of Nimvec AM510 toward providing life-changing treatments for patients in need."