Sensorion’s SENS-601 strategy signals a sharper bet on larger genetic hearing loss markets
Find out how Sensorion’s SENS-601 regulatory push could reshape GJB2 hearing loss gene therapy and clinical strategy.
Can salanersen help Biogen defend its rare neurology franchise beyond Spinraza?
Find out how Biogen’s salanersen could reshape spinal muscular atrophy treatment after FDA breakthrough therapy status.
A rare disease policy shift just landed at the WHO. Could it change hemophilia care worldwide?
Find out how the WHO hemophilia resolution could reshape diagnosis, treatment access, prophylaxis and bleeding disorder care worldwide.
Eli Lilly expands genetic medicines toolkit with Engage Bio’s non-viral DNA delivery system
Eli Lilly’s Engage Bio deal is small by its standards, but DNA delivery may become a big battleground in genetic medicines.
Can Ferring turn real-world ADSTILADRIN use into a broader NMIBC treatment strategy?
Bladder-sparing therapy needs better sequencing. Ferring’s ADSTILADRIN re-induction signal raises hope, but durability is the real test.
Can non-viral gene therapies solve the safety problem holding back genetic medicine?
Viral vectors built gene therapy’s first era. Safety, redosing and manufacturing pressure are pushing non-viral platforms into the spotlight.
Why AAVnerGene’s AAVone 2.1 could matter for next-generation gene therapy manufacturing
AAV gene therapy needs better economics. AAVnerGene’s AAVone 2.1 tests whether single-plasmid production can ease the scale-up squeeze.
FDA RMAT status puts Rznomics’ RNA editing platform under a brighter clinical spotlight
RNA editing just gained a liver cancer spotlight. Rznomics must now prove RZ-001 can turn early response signals into durable clinical value.
How CPTx is positioning targeted lipid nanoparticles at the center of CAR T innovation
Explore how CPTx’s targeted lipid nanoparticle platform could reshape CAR T therapy scalability and access. Read the full analysis now.
Why Spur Therapeutics’ gene therapy data signals a shift in Gaucher disease bone management
New FLT201 data shows potential bone disease improvement in Gaucher patients. Find out what this means for future treatment strategies.