Around the Helix: Cell and Gene Therapy Company Updates – August 16, 2023


Catch up on the latest news, breakthroughs, and announcements from biotechnology companies making advancements in cell and gene therapies.

CGTLive Around the Helix

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 Places Formal Clinical Hold On 2seventy bio's Trial for Acute Myeloid Leukemia CAR-T In Relation to Patient Death

The FDA has placed a formal clinical hold on the phase 1 PLAT-08 clinical trial (NCT05105152) evaluating 2seventy bio’s SC-DARIC33, an investigational CD33-directed chimeric antigen receptor T-cell (CAR-T) therapy intended to treat acute myeloid leukemia, in relation to the death of the first patient treated at the study’s second dose level (5x106 SC-DARIC33 T-cells/kg).

2. Parkinson Disease iPSC-derived Therapy ANPD001 Cleared for Clinical Trial in the US

Aspen Neuroscience has received clearance from the FDA for an investigational new drug application for ANPD001, an investigational autologous induced pluripotent stem cell (iPSC) derived therapy intended to treat Parkinson disease. ANPD001 consists of dopamine neuronal precursor cells produced from iPSCs that are in turn derived from samples of patients’ own skin cells.

3. FDA Lifts Partial Clinical Hold on Arcellx’s Trial for Multiple Myeloma CAR-T CART-ddBCMA

The FDA has lifted its partial clinical hold on the phase 2 IMMagine-1 clinical trial (NCT05396885) evaluating CART-ddBCMA, an investigational autologous CAR-T therapy intended to treat relapsed/refractory multiple myeloma. The partial clinical hold was originally placed in June 2023 in relation to the death of a patient participating in the trial.

4. Genprex Garners Orphan Drug Designation for Lung Cancer Immunogene Therapy Reqorsa

Genprex’s quaratusugene ozeplasmid (Reqorsa), an investigational immunogene therapy intended to treat various forms of lung cancer, has been granted orphan drug designation by the FDA for the treatment of extensive-stage small cell lung cancer in combination with Genentech's checkpoint inhibitor atezolizumab (Tecentriq) for patients who did not experience tumor progression following initial standard treatment with atezolizumab and chemotherapy.

5. American Gene Technologies and 10X Capital Venture Acquisition Corp. III Enter Combination Agreement

The newly combined company will operate under the name Addimmune, will be publicly traded, and will use “HIV” as a ticker symbol. The new company will continue to develop American Gene Technologies (AGT)’s investigational HIV gene therapies and cell therapies, but will move development of nonHIV investigational products to a spinout company that will operate under the AGT name.

6. Henrietta Lacks Estate Sues Ultragenyx

Civil rights and personal injury lawyer Ben Crump has filed a lawsuit on behalf of Henrietta Lacks’ estate against gene therapy company Ultragenyx over the use of the HeLa cell line in the production of its adeno-associated virus (AAV) vector-based gene therapies. The immortal HeLa cell line is derived from cells taken from Henrietta Lacks without her permission by doctors at Johns Hopkins Hospital in the 1950s during her treatment for cervical cancer. “Ultragenyx’s choice to continue utilizing HeLa cells despite the cell line’s origin and the concrete harm it inflicts on the Lacks family can only be understood as a choice to embrace a legacy of racial injustice embedded in the US research and medical systems,” Crump said in a statement.

7. GenScript Biotech to Supply T-MAXIMUM Biotech With CRISPR Nucleic Acid Reagents 

The CRISPR reagents will aid T-MAXIMUM in its development of its investigational CAR-T therapies, which include MT027, a universal CAR-T intended to treat recurrent high-grade gliomas. “We are delighted to establish a strategic cooperation with GenScript...” Shang Xiaoyun, PhD, the CEO of T-MAXIMUM, said in a statement. “Gene editing reagents are crucial raw materials for the quality and efficacy of our products. We are confident that this collaboration will accelerate the regulatory processes in China and the United States, expediting T-MAXIMUM’s first product pipeline to benefit patients in need as soon as possible.”

8. Neurophth Rakes in Close to $95 Million USD in Series C+ Round

The Chinese company is focused on the development of gene therapies for ophthalmic indications and has a pipeline that includes NR082, an investigational gene therapy for Leber's hereditary optic neuropathy that is being evaluated in the first phase 3 gene therapy clinical trial to have finished enrollment in China.

9. Precision Biosciences Sells Off CAR-T Program to Imugene

Precision Biosciences has sold global rights to Imugene for azercabtagene zapreleucel (azer-cel), a clinical-stage allogeneic CAR-T therapy being evaluated for the treatment of large B-cell lymphoma in patients whose disease relapsed after previous treatment with autologous CAR-T. In light of the completion of the strategic transaction, Precision intends to shift its focus entirely to its ARCUS in vivo gene editing programs.

10. Newly Formed CRDMO GeneFab Subleases Senti Biosciences’ Manufacturing Facility in Alameda, California

New contract research, development, and manufacturing organization (CRDMO) GeneFab simultaneously announced its launch and the deal with cell therapy and gene therapy developer Senti Biosciences that also includes a purchase o fSenti Bio’s chemistry, manufacturing, and controls capabilities and the addition of Senti Bio’s departing chief technology officer Philip Lee, PhD, as CEO of GeneFab.

11. Mass Photometry Instruments Manufacturer Refeyn Announces AAV Analytics Software Package

The software package for SamuxMP mass photometers is intended to allow gene therapy manufacturers to measure empty to full AAV ratios as a quality control method in good manufacturing practice (GMP) environments. "Refeyn's new software package for GMP enables the seamless integration of mass photometry into AAV manufacturing processes, unlocking the efficiency that SamuxMP instruments bring to AAV analytical workflows," Matthias Langhorst, the chief product officer of Refeyn, said in a statement.

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