Around the Helix: Cell and Gene Therapy Company Updates – July 12, 2023

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. AVROBIO Ceases Development on All Programs

AVROBIO has announced a cessation of all development activities in order to evaluate its strategic options for the future, which the company noted may include seeking an acquisition, merger, business combination, or another type of transaction. The announcement comes just a month after AVROBIO sold its cystinosis hematopoietic stem cell (HSC) gene therapy program to Novartis. AVROBIO had also been developing HSC gene therapies for indications including Gaucher disease type 1 and type 3, Hunter syndrome, and Pompe disease.

2. HEMO-CAR-T IND Gets Clinical Hold for Manufacturing Issue

The FDA has placed Hemogenyx’s HEMO-CAR-T investigational new drug application (IND) for treating acute myeloid leukemia on clinical hold and has provided details about the hold in a full review letter. In the letter, the FDA shared the reasoning behind the hold, which relates to a splicing that occurs during the manufacturing process of the lentivirus vector. Hemogenyx stated that it has identified the source of the splicing issue, has developed a method to eliminate it, and is remanufacturing the lentivirus.

3. RNA Therapy Shows No Benefit for ALS or FTD

WVE-004 did not show clinical benefit in treating patients with amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD). Wave Life Sciences will discontinue development of the RNA therapy. The company announced topline results from the phase 1b/2a FOCUS-C9 study (NCT04931862) evaluating WVE-004 as an investigational treatment for C9orf72-associated ALS and FTD.

4. Sensorion Submits UK Clinical Trial Application for OTOF Hearing Loss Gene Therapy

Sensorion has submitted a clinical trial application (CTA) to the UK’s Medicines and Healthcare products Regulatory Agency for OTOF-GT, an investigational gene therapy intended for the treatment of otoferlin (OTOF) gene-mediated hearing loss. Pending clearance of the CTA, the company intends to initiate the planned phase 1/2 Audiogene clinical trial, which will evaluate OTOF-GT in patients aged 31 months and younger. Sensorion stated that it has successfully manufactured the gene therapy product for use in the clinical trial.

5. First Patient With NHL Receives γδ T-Cell Therapy in Phase 1 Trial

Acepodia has dosed the first patient in the phase 1 trial (NCT05653271) evaluating ACE1831 for the treatment of non-Hodgkin lymphoma (NHL). ACE1831 is a γδ T-cell therapy developed with the use of Acepodia’s Antibody-Cell Conjugation platform and is the company’s first therapy of this kind to enter clinical trials. The therapy targets CD20-expressing hematological cancers and has demonstrated enhanced cytotoxicity against cancer cells both in vitro and in vivo in animal models.

6. Pfizer Invests $25 Million in Caribou Biosciences

In addition to the purchase of common shares, Pfizer has added Sriram Krishnaswami, PhD, the vice president and development head for Multiple Myeloma at Pfizer Oncology’s Global Product Development division, to Caribou Biosciences’ Scientific Advisory Board. “We are encouraged by Caribou’s chRDNA genome-editing technology and the potential of allogeneic cell therapies as a promising off-the-shelf approach to cancer treatment,” Krishnaswami said in a statement.

7. Genprex Adds Patent Covering Reqorsa-Keytruda Combination Therapy in China to Its Repertoire 

Genprex is currently evaluating its Reqorsa Immunogene Therapy (quaratusugene ozeplasmid) in combination with the PD1 antibody Keytruda in the phase 1/2 Acclaim-2 clinical trial (NCT05062980) for the treatment of advanced, metastatic non-small cell lung cancer. The Chinese patent is the latest in a series of patents covering the use of Reqorsa with PD1 antibodies that Genprex has garnered incountries including the United States, Japan, Australia, Russia, and Mexico.

8. Precision Biosciences Assigns Eli Lilly Subsidiary Prevail Therapeutics Additional Heavy Lifting in Collaboration Amendment

According to a June 30, 2023, SEC filing, a previously established collaboration agreement between Prevail Therapeutics, a subsidiary of Eli Lilly and Company, and Precision Biosciences, has been amended with additional responsibilities added for Prevail. These include Prevail conducting and funding gene therapy preclinical research that was previously covered by Precision and Prevail performing manufacturing of initial clinical trial material for the first licensed product in the collaboration.

9. Tessa Therapeutics Is No More

Tessa Therapeutics, a Singapore-based company that had been developing cancer cell therapies, has closed its doors, according to a letter to shareholders reported on by The Business Times that attributed the shutdown to an inability to secure funding. Notably, Tessa’s website is currently inaccessible and displays a message indicating it is under maintenance.

10. Astellas Pharma Buys Rights to use AAV Vector R100 from 4D Molecular Therapeutics

R100, which was developed by 4D Molecular Therapeutics and is used in its clinical-stage ophthalmic gene therapies, is an adeno-associated virus (AAV) vector intended to deliver genetic payloads to retinal cells across the internal limiting membrane barrier. Astellas has bought the right to use it for targeting 1 rare monogenic ophthalmic disease; under the agreement, the company can also purchase options to use it in 2 more rare monogenic ophthalmic disease indications.

11. Kytopen Adds Flowfect Discover Gene Editing Instrument and Flowfect Tx Manufacturing-scale Platform to Rice University’s Omid Veiseh Laboratory

The Flowfect Discover and Flowfect Tx technologies will be utilized in the lab’s research aimed at developing new cell therapies for various indications. “By equipping researchers at the forefront of cell therapy discovery at Translational Medical Research Centers with Flowfect platforms, we aim to revolutionize the field of cell therapy discovery, reduce costs associated with scaling and manufacturing, and ultimately improve patient access to the next generation of life saving medicines,” Greg Crescenzi, the chief commercial officer of Kytopen, said in a statement.

12. First Engineering Batch in US for Nexcella’s AL Amyloidosis CAR-T Finished, Clearing Way for Clinical Expansion

NXC-201 (HBI0101) is an investigational chimeric antigen receptor T-cell (CAR-T) therapy currently being evaluated in the phase 1b/2a NEXICART-1 clinical trial (NCT04720313) at Hadassah University Hospital in Israel. “The completion of our first US engineering batch is a significant step towards expanding our phase 1b/2a NEXICART clinical trial to the United States as we work to bring NXC-201 to American patients in need,” Ilya Rachman, MD, PhD, the executive chairman of Nexcella, said in a statement.