Around the Helix: Cell and Gene Therapy Company Updates – October 9, 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. First Patient Dosed Achieves Complete Response in Estrella Immunopharma’s Trial for CD19-Targeted T-cell Therapy EB103 in B-Cell Lymphomas

The first patient dosed in Estrella Immunopharma’s phase 1/2 STARLIGHT-1 clinical trial (NCT06343311) evaluating EB103, an investigational autologous CD19-targeted T-cell therapy, for the treatment of B-cell lymphomas has achieved a complete response.

2. Immix and Nexcella Make Progress in US Trial for Light Chain Amyloidosis CAR-T NXC-201

The phase 1b/2 NEXICART-2 clinical trial (NCT06097832), which is evaluating Immix Biopharma and its subsidiary Nexcella's NXC-201, an investigational autologous chimeric antigen receptor T-cell (CAR-T) therapy, for the treatment of relapsed/refractory (r/r) light chain amyloidosis has moved onto its expansion cohort after completing dosing of patients in its initial cohort.

3. In Light of Positive Initial Results, Ultragenyx Seeks to Add Higher Dose Cohort to Trial for Wilson Disease Gene Therapy UX701

Ultragenyx intends to submit a protocol amendment for the phase 1/2/3 Cyprus2+ clinical trial (NCT04884815) evaluating UX701, an investigational adeno-associated virus (AAV) vector-based gene therapy intended to treat Wilson disease, in order to evaluate a higher dose cohort and optimized immunomodulation regimen.

4. Anixa Biosciences Seeks Protocol Change to Enable Repeat Dosing in Trial for Ovarian Cancer CER-T

Anixa Biosciences and its partner Moffitt Cancer Center have submitted a protocol amendment to the FDA for their ongoing phase 1 clinical trial (NCT05316129) evaluating Anixa's novel follicle stimulating hormone receptor (FSHR)-targeting chimeric endocrine receptor (CER) T-cell therapy for the treatment of ovarian cancer, aimed at enabling the use of second doses for patients who may benefit.

5. Aspen Neuroscience and Cell X Team Up to Automate iPSC Manufacturing

Aspen Neuroscience and Cell X Technologies have signed a collaboration agreement aimed at automating the induced pluripotent stem cell (iPSC) stage of the former's autologous manufacturing platform with the use of the latter's Celligent platform. "In order to treat greater numbers of patients with their own cells, Aspen is developing a robust, scalable, and automated biomanufacturing system," Kim Raineri, the chief technology officer of Aspen Neuroscience, said in a statement. "We have worked with the Celligent platform and believe that, combined with our bioinformatics and other enabling technologies, it has the potential to successfully automate iPSC production."

6. GEMMABio and Fiocruz Join Forces to Bring Gene Therapy to Brazilian Patients

GEMMA Biotherapeutics (GEMMABio) and the Oswaldo Cruz Foundation (Fiocruz) of the Ministry of Health of Brazil have entered an agreement that will involve Fiocruz providing up to $100 million in funding for GEMMABio to carry out research and manufacturing activities aimed at bringing accessible gene therapy treatments to Brazilian patients. "By combining our resources and knowledge, this collaboration represents a major advancement in making gene therapies accessible to more patients quickly and at a fraction of the typical cost," Jim Wilson, MD, PhD, the founder of GEMMABio, said in a statement.

7. MiNK Therapeutics and Autonomous Therapeutics Aim to Tackle Metastatic Tumors With Combined Technology

MiNK Therapeutics is seeking to combine its allogeneic invariant natural killer T (iNKT) cell therapy technology with Autonomous Therapeutics' precision encrypted RNA (encRNA) technology in order to develop new treatment options for metastatic tumors. “This exciting collaboration leverages the platform capabilities of our next-generation encrypted RNA technology and MiNK’s unique allogeneic cell therapy platform," Ariel Weinberger, PhD, the CEO of Autonomous, said in a statement. "AT313 is a preclinical encRNA candidate developed at Autonomous to enable the precision targeting of solid tumors, via cancer-activated therapeutic protein translation. Our aim is to develop novel medicines that pinpoint tumor cells in patients—by sensing and targeting the aberrant molecular signatures that define cancer replication."

8. BioHub Maryland Obtains Grant to Build Up Gene Therapy Manufacturing Workforce

The Maryland Department of Labor has provided a $75,000 EARN Maryland grant to BioHub Maryland for the purposes of training citizens in the state in bioprocessing skills relevant for the manufacture of several types of medical products, including cell and gene therapy products. "The life sciences sector is a major driver of Maryland's economy, with 54,000 workers in Maryland," Kelly Schulz, the CEO of the Maryland Tech Council, said in a statement. "With a national shortage of biopharma talent, we have a clear opportunity to enhance Maryland’s competitive edge by reskilling current employees and upskilling newcomers."