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The advanced therapies news this week includes a breakthrough in autoimmune care using allogeneic CAR T cell therapy and FDA approval of the first genetically modified T cell therapy for treating a rare sarcoma.
Welcome to Phacilitate’s weekly news round-up, which will bring you the latest advanced therapies news and resources each Friday in the lead up to Advanced Therapies Week 2025 in Dallas. This round-up covers manufacturing, investment, therapeutic development, clinical trials, and any stories making the headlines that week.
The news this week includes a breakthrough in autoimmune care using allogeneic CAR T cell therapy and FDA approval of the first genetically modified T cell therapy for treating a rare sarcoma.
If you have any news you’d like to share, or if you’d like to add additional commentary, please get in touch.
Read on to find out what else has been happening this week.
Headlines
Our first news headline will be a welcome one for patients with autoimmune conditions. Three patients have gone into remission from severe autoimmune conditions after receiving donor-derived CAR T-cell therapy. If treatment succeeds in more people over a longer time frame, it “could prove paradigm shifting”, says Daniel Baker, an immunologist at the University of Pennsylvania in Philadelphia.
The FDA’s approval of Afamitresgene autoleucel (Tecelra) makes it the first engineered T cell therapy to gain approval for a solid tumor indication. This opens up a range of therapeutic options for TCR-T cells.
Now onto the hot topic of artificial intelligence (AI). TC BioPharm has entered into a sponsored research agreement with Carnegie Mellon University to explore the potential use of AI to optimize the patient screening process.
Bryan Kobel, CEO of TC BioPharm, explained, “Finding a solution for how to select the best donors for allogeneic cell therapies to generate the best product, and also how to match the best cell therapy and donor to the best patient beyond the current HLA matching process, represents a massive opportunity from a market perspective and also would have far-reaching applications for CDMOs and other industry players.”
Therapeutic Development and Clinical Trials News
Intellia Therapeutics, a clinical-stage gene editing company has initiated a global Phase 3 study of NTLA-2002 for the treatment of hereditary angioedema (HAE). The HAELO trial is a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of NTLA-2002 in 60 adults with Type I or Type II HAE.
The National University of Singapore (NUS) has announced that its fratricide-resistant CD7 CAR-T therapy has proven effective in treating relapsed or refractory T-cell leukaemia. The therapy was administered to 17 patients with 16 of the patients observed achieving complete remission within one month.
Aurigene Oncology Limited, which is a wholly-owned subsidiary of Dr. Reddy’s Laboratories, has announced the Phase 1 results for India’s first trial for a novel autologous BCMA directed CAR-T cell therapy in patients with relapsed / refractory multiple myeloma. All eight patients achieved clinical response, with five out of eight having achieved stringent complete response.
Commercialization and Investment News
Purespring Therapeutics, a gene therapy company focused on treating kidney diseases, has raised £80/$105 million in Series B financing. Proceeds will be used to support Purespring’s pipeline, including the initiation of a Phase I/II clinical trial for IgAN, a common, chronic kidney disease primarily affecting young adults.
GEMMABio, a new therapeutics company founded by gene therapy pioneer Dr. Jim Wilson, will receive up to $100 million of funding as part of an agreement with Brazilian public health research institution, Oswaldo Cruz Foundation.
Astellas Pharma has received an exclusive option to license AviadoBio’s investigational, AAV-based gene therapy, which is in Phase 1/2 development for patients with frontotemporal dementia with progranulin mutations. AviadoBio will receive $20 million equity investment, up to $30 million in upfront payments, and is eligible to receive up to $2.18 billion in license fees and milestone payments plus royalties.
Manufacturing News
OpenCell Technologies is to integrate its Softporation technology with Adva Biotechology’s ADVA X3 platform to create an end-to-end manufacturing solution for the rapidly evolving bioprocessing market.
Ginkgo Bioworks has partnered with Virica Biotech to enhance their adeno-associated virus (AAV) gene therapy manufacturing platforms. Ginkgo will leverage their advanced screening platform for rapid testing of VSEs directly in clients’ cell lines. Virica will provide a panel of their cell enhancers for use in high-throughput AAV production screening for Ginkgo’s clients.
Cell X Technologies, and Aspen Neuroscience have inked a licensing agreement focused on providing an automated solution for Aspen’s iPSC-derived autologous clinical manufacturing. The two companies are committed to accelerating manufacturing development for the benefit of people suffering with Parkinson’s disease.
Reports
The biopharmaceutical processing equipment and consumables market is expected to grow 9.5% in the period of 2024-2031 to reach $75.72 billion by 2031. The growth will be driven by the growing biopharmaceuticals market, biopharmaceutical plant capacity expansions, and the rising adoption of single-use bioprocessing equipment, as well as potentially emerging economies, the shift toward Bioprocessing 4.0, and the rising adoption of personalized medicines.
The cell and gene therapy manufacturing services market is expected to grow from $11.4 billion in 2023 to $70.7 billion by 2032, which is a growth rate of 22.4%.
If you want to continue the discussion, why not register for Advanced Therapies Week 2025 in Dallas, the must-attend event for the biotech ecosystem.
