Rare & Genetic Diseases
| 4 min read
At the Advanced Therapies conference, industry leaders showcased how in vivo CAR T cell and gene delivery innovations could transform accessibility and clinical outcomes.
| 4 min read
From early experiments in rare genetic diseases to next-generation tools that can edit, insert, and regulate genes, gene therapy is transforming what’s possible for patients.
| 5 min read
Industry leaders at the Advanced Therapies conference outline how early collaboration, funding, and culture shape whether breakthroughs reach patients.
| 5 min read
A new in vivo approach programs patients’ T cells directly, bypassing complex manufacturing and expanding access to CAR T therapies.
| 6 min read
Fit-for-purpose analytics like mass photometry give teams the confidence to refine processes before scaling to GMP-compliant production.
| 4 min read
Unexpected breakthroughs in drug manufacturing, lawsuits, regulatory updates, and more led the news this week.
| 5 min read
Ireland’s model for advanced therapy manufacturing integrates academic research, workforce development, and regulatory bodies to streamline the production of complex biologics.
| 3 min read
A new biological foundation model analyzes genomic patterns across the tree of life and may accelerate disease research and synthetic biology.
| 3 min read
When science, manufacturing, and regulatory alignment move together, breakthroughs reach patients faster than ever before.
| 5 min read
With gene therapy now a clinical reality, innovators are designing delivery systems that can sustain lifelong correction, handle complex genes, and make treatments accessible to more patients.
| 4 min read
Biopharma’s recovery is real, but the data show a sector that now rewards validated biology, late-stage execution, and commercial relevance.
| 3 min read
PolyActiva and RareSight are pairing pro-drug chemistry with targeted ocular delivery to address early-stage barriers in rare pediatric retinal disorders.
