Pharmaceuticals & Therapeutics

ProQR Therapeutics secures a crucial $59.2M, extending its runway and gaining a powerful endorsement from partner Eli Lilly for its Axiomer RNA editing platform.

ProQR's Axiomer platform achieves its first human validation, showing promise for rare pediatric liver diseases and signaling a major step for RNA editing.

Insmed and EMBARC's 3,000-patient study of brensocatib aims to do more than treat a chronic lung disease—it aims to fundamentally alter its course.

ARS Pharmaceuticals' needle-free allergy spray, neffy, faces a major setback with commercial insurers, forcing a strategic pivot toward fiscal discipline.

The FDA has approved TRYNGOLZA, the first drug to reduce triglycerides and acute pancreatitis risk, marking a new era for millions and a pivotal moment for Ionis.

A small biotech reports its novel curcumin gel eases sickle cell pain. But its strategy of pre-approval access via pharmacies is a high-stakes economic gamble.

A landmark deal brings VIZZ, a once-daily presbyopia treatment, to Australia and New Zealand, promising millions a life with less reliance on reading glasses.

REPROCELL's submission for Stemchymal® offers hope for an incurable disease and highlights Japan's accelerated regulatory path for regenerative medicine.

Heparin, a trusted blood thinner, can trigger a fatal immune reaction. A small biotech's novel drug is now taking the global stage with a targeted solution.

Imbria's ninerafaxstat gains FDA Orphan Drug status for nHCM, a rare heart condition. This strategic win pits its unique metabolic approach against rivals.

A key FDA alignment for zervimesine not only de-risks a pivotal trial but signals a new era for treating the devastating psychosis of an overlooked dementia.

BOTOX® Cosmetic is investing millions in women entrepreneurs. Is it smart philanthropy, or a brilliant new blueprint for brand building and corporate strategy?

An alliance of biopharma innovators is set to discuss a critical hurdle in medicine: delivering RNA therapies to tissues beyond the liver, a key to unlocking new treatments.

A new report shows AI directs 80% of aesthetic medicine searches to AbbVie & Galderma, creating a 'visibility gap' that leaves 11,500 small clinics invisible.

With a $2.74M grant from The Michael J. Fox Foundation, biotech startup X-tosis is advancing a novel therapy that aims to fix the cell's failing powerhouses.

With a key grant from Breakthrough T1D, MannKind aims its inhaled insulin at children. Is this a life-changing innovation or a high-stakes corporate revival?

Quoin Pharma's new drug name is a key step, but the path to the first-ever treatment for a rare skin disease reveals the complex interplay of innovation and finance.

A novel drug aims to reverse myelin damage in MS, a feat no current therapy can achieve. But with a recent FDA hold, is Quantum BioPharma's vision a breakthrough or a mirage?

BioMendics secured a key FDA Fast Track designation for its EBS therapy, a strategic move that de-risks development and signals a new era for rare disease treatment.

With a novel nasal spray targeting the host, not the virus, RA Capital-backed NorthLinks Bio aims to create a broad-spectrum defense against respiratory threats.