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Gene Therapy
As India prepares to commercialize its first advanced therapies it must engage key stakeholders to ensure successful implementation. Learning from global best practices and managing pricing strategies will be crucial to maximize access, deliver high-quality outcomes, and sustain innovation through reinvestment in R&D.
Circular RNA has several advantages, but the field is young. Scandinavian startup Circio Holdings believes its version of the technology will prove the most robust.
Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).
Beam presented data from three more patients than during its last update for BEAM-101, showing stem cell engraftment and efficacy consistent with the first four patients.
Experts working in the advanced therapy sector tell In Vivo how novel solutions can empower cell and gene therapy manufacturers to reduce costs, improve scalability and optimize their processes – improving the clinical profile and commercial viability of products.
A wealth of new therapies are set to successfully graduate from the pipeline in 2025. Within this cohort are a mixture of therapeutic areas, drug classes, first-time approvals, label expansions, and treatments that will meaningfully change how diseases are treated.
Big pharma executives at ARM’s Cell and Gene Meeting on the Mesa shared some of the practical challenges of bringing one-time treatments to market in hemophilia, cancer and beyond.
Trevor Martin talked to In Vivo about the importance of sharing a strategic vision with partners, the company’s superpower, and how a PhD is the best prep for a startup.
With more advanced therapies gaining approval, companies at ARM’s Cell and Gene Meeting on the Mesa considered hurdles to making their products more broadly available and developing them for larger indications.
Purespring has just raised $105m from an investor syndicate who are betting it can develop the first gene therapy for kidney disease and learn from setbacks experienced in the space.