Around the Helix: Cell and Gene Therapy Company Updates – June 7, 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.

1. New Clinical Data Presented at ASCO 2023

Catch up on CGTLive’s coverage of the American Society of Clinical Oncology (ASCO) 2023 Annual Meeting, held June 2-6, in Chicago, Illinois. Our coverage will continue throughout the week.

2. PepGen’s Myotonic Dystrophy Oligonucleotide Trial Stalls

The FDA has placed a clinical hold on PepGen’s investigational new drug application for a phase 1 study of PGN-EDODM1, an enhanced delivery oligonucleotide for the potential treatment of Myotonic Dystrophy Type 1. An official clinical hold letter that will detail the reasons for the hold will be sent by the FDA within 30 days.

3. Trial for Rett Syndrome Gene Therapy TSHA-102 Doses First Patient

The first patient has been dosed in the phase 1/2 REVEAL clinical trial (NCT05606614) evaluating Taysha Gene Therapies’ TSHA-102, an investigational adeno-associated virus (AAV) vector-based gene therapy, for the treatment of adult patients with Rett syndrome. TSHA-102, which is administered intrathecally, delivers a copy of miniMECP2, a truncated version of the disease-targeted MECP2 gene, via an AAV9 vector.

4. FDA Clears Cabaletta Bio’s CAR-T Agent CABA-201 for Trial in Myositis

Cabaletta Bio’s CABA-201, an investigational CD19-directed chimeric antigen receptor T-cell (CAR-T) therapy intended to treat autoimmune diseases, has received clearance of its investigational new drug application by the FDA for a clinical trial in patients with active idiopathic inflammatory myopathy, also referred to as myositis. CABA-201 contains a 4-1BB costimulatory domain and is intended to deplete B-cells in order to “reset” the immune systems of patients.

5. InnDura Therapeutics Enlists the Aid of Landmark Bio for NK Cell Program

InnDura Therapeutics’s lead program incorporates EVE16 engineering of natural killer (NK) cells, a technology which derives from an exclusive license agreement with Dana-Farber Cancer Institute and the Massachusetts Institute of Technology. The technology is intended to improve the cancer-killing abilities of NK cells while also decreasing cellular exhaustion. Landmark Bio will provide chemistry, manufacturing, and control and other support to streamline further development of the program.

6. Cellular Origins Unveils Robotic Cell Therapy Manufacturing Platform 

Constellation, which incorporates the use of mobile robots and automated tube management, is intended to allow for cell therapy manufacturing at scale without the need to alter processes used in clinical trials. The company stated that in comparison to conventional manufacturing methods the platform is capable of improving space-efficiency by 30-fold.

7. BioRestorative Therapies to Leverage Licensing as Tissue Bank in New York State 

BioRestorative Therapies has announced its intention to utilize its new license from the New York State Department of Health to act as a tissue bank to bank its lead cell therapy product BRTX-100 and to provide new cashflow by offering contract manufacturing services to other companies.

8. Rentschler Biopharma to Provide CDMO Services for Ikarovec’s Investigational Ophthalmic Gene Therapy

Ikarovec’s novel gene therapy, which is intended to treat geographic atrophy, is currently in the preclinical stage of development. “As we look to find new and better ways to treat ophthalmic diseases, we want to get it right and ensure that our product candidates can be scaled up appropriately for each development stage and, ultimately, for commercialization,” Katie Binley, the chief scientific officer of Ikarovec, said in a statement.

9. Mission Bio Updates Its Tapestri Platform

Tapestri v3, the latest iteration of the Tapestri Platfrom, which is intended to allow for detection of rare cells, is expected to allow for a greater number of cells to be captured in samples. Mission Bio noted that the platform could help to identify new targets for therapeutics and improve evaluation of metrics for risk of disease relapse.

10. CDMOs RoslinCT and Lykan Bioscience Merge

The newly combined company will be referred to under the RoslinCT name and will offer CDMO services to companies developing cell therapies and gene therapies. “We are thrilled about the integration of 2 leading organizations in the advanced cell and gene therapy space, resulting in 22 cGMP processing suites, enhanced expertise, resources, and transatlantic cGMP manufacturing capabilities to support the development and commercialization of advanced cell and gene therapies worldwide,” Patrick Lucy, the president and CEO of Lykan Bioscience, said in statement.

11. StemInov Announces Production of MSC Therapy for Inflammatory Diseases

The company’s mesenchymal stem cell (MSC) therapy is intended to treat inflammatory diseases through modulation of inflammation and antibacterial activity. Referred to as WhartSep, it is composed of MSCs derived from Wharton’s Jelly in the umbilical cord; the company expects that it will be able to target damaged organs.

12. Summation Bio Closing Down

The biotech start-up, which was operating in stealth and working on a novel nonviral gene therapy technology, will be ceasing its operations. The announcement came in the form of a LinkedIn post from senior director and head of program management Chris Hanff, who cited “circumstances insurmountable” as a reason for the decision.