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Welcome to Phacilitate’s weekly cell and gene therapy (CGT) news round-up, which will bring you the latest advanced therapies developments each Friday in the lead up to Advanced Therapies Week 2025 in Dallas.
This round-up covers therapeutic development, clinical trials, technology, and product news and any stories making the headlines that week.
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.
Advanced Therapies Developments: Headline of the Week
The headline this week is regarding the mainstream recognition of the impact of cell and gene therapies and the incredible work that goes in to developing these drugs.
In December 2023, Vertex Pharmaceuticals and CRISPR Therapeutics Casgevy became the first CRISPR-based therapy to receive FDA approval. Time magazine has now recognized the therapy as one of the best inventions of 2024.
Clinical Trial News
The FDA has cleared RiboX Therapeutics’ IND application for the Phase 1/2a Study of RXRG001, which is the first-ever circular RNA therapy to receive FDA IND clearance. The first-in-human SPRINX-1 study is designed to evaluate the safety and efficacy of RXRG001 in patients with radiation-induced-xerostomia (dry mouth) and hyposalivation (low saliva secretion).
PolTREG has launched a placebo-controlled Phase 2 clinical trial with PTG-007 Treg cell therapy in presymptomatic type-1 diabetes (T1D) patients, after receiving approval from the European Medicines Agency. The study will evaluate safety and efficacy in 150 patients, including those aged between 6 and 16 years who are at high genetic risk of developing T1D, but who have not yet shown any symptoms.
Therapeutic Developments
As Pharmaceutical Technology reports, Dyno Therapeutics has partnered with Roche to develop adeno-associated virus (AAV) gene therapy vectors to target neurological diseases. Dyno Therapeutics will develop novel AAV capsids by incorporating AI and high-throughput in vivo data collection. Roche will conduct capsid validation studies into further preclinical, clinical, and commercialization activities for the gene therapy assets.
Technology News
NewBiologix has launched its Xcell suite of technologies to address challenges in gene therapy production, such as low yields, batch heterogeneity, scalability, and high cost, as well as provide insights and comprehensive analysis of rAAV quality.
Product News
Parse Biosciences has launched its GigaLab scalable single cell sequencing solution. The GigaLab was built for researchers scaling to projects of 10 million cells and more. According to the company, the new capabilities will help researchers focused on large-scale drug and perturbation screens, dataset generation for generative AI models, and atlasing for population studies.
Mergers and Acquisitions
Lyell Immunopharma is to acquire ImmPACT Bio, a privately-owned clinical-stage biotechnology company. Lyell will continue to develop ImmPACT’s lead program, IMPT-314, which is a CD19/20-targeting CAR T-cell product candidate for hematologic malignancies, including large B-cell lymphoma.
Industry Insights
Pharma Voice has published its Cell and Gene Therapy Pioneers for 2024. This year’s top voices come from a mixture of companies, including pharma leaders such as Regeneron and Vertex along with newer therapeutic developers and start-ups.
The Association for the Advancement of Blood & Biotherapies (AABB) has introduced the proposed first edition of Cell and Gene Therapy Standards for Pharmacy. The AABB standards will guide pharmacies in the receipt, storage, handling and dispensing of these therapies. A review of existing pharmacy quality programs and available certifications revealed a lack of standards for handling and managing CGTs within pharmacy.
Reports
The global cell and gene therapy market is anticipated to grow at a rate of 25.8% from 2023 to 2031. In real terms, the market was worth US$ 4,485.00 million in 2023 and is expected to reach US$ 28,050.39 million by 2031.
According to a new Treg cell-based therapies pipeline report, there are more than 50 active players working in the space developing more than 55 pipeline therapies for various indications. Treg cell-based therapies focus on harnessing regulatory T cells (Tregs) to modulate the immune response in various clinical settings.
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.
