Gene Therapies

A groundbreaking partnership between Servier and n-Lorem Foundation blends pharma R&D muscle with non-profit agility to develop ASO therapies for the rarest of diseases.

Sanofi's promising nerve disease drug fails in late-stage trials, leaving vulnerable patients in limbo and highlighting the brutal realities of medical innovation.

A novel TYK2 inhibitor that can enter the central nervous system has begun human trials, signaling a new frontier for treating diseases like MS and lupus.

New long-term data for masitinib shows a near doubling of 5-year survival for ALS patients, with many maintaining quality of life. Is this the turning point?

A $20M round for CELYSTRA Pharma isn't just a launch; it's a strategic maneuver to solve Canada's rare disease gap and redefine Québec's role in the market.

A Maryland firm just got funding for 'universal' stem cells. This isn't just science; it's the blueprint for off-the-shelf cures and a new era of medicine.

With a novel 'gastric bypass in a pill' and a new CFO known for orchestrating lucrative exits, Syntis Bio is gearing up for a high-stakes battle in the booming obesity drug market.

ResVita Bio's new board member signals a major shift. We explore how visionary leadership and living medicine are converging to tackle long-neglected diseases.

New research from Baycrest unmasks paratonia, an involuntary condition often mistaken as defiance, signaling a seismic shift in dementia care and treatment.

As Capricor presents its late-stage DMD drug and novel exosome platform, can it convince investors it has the key to both patient impact and market success?

With expanded coverage for 52 million Americans, Tonix Pharmaceuticals' TONMYA® is not just a drug launch; it's a market disruption for chronic pain.

As GLP-1 drugs dominate, a UK biotech reveals a new drug that targets mitochondria, promising superior fat loss while preserving precious muscle mass.

Zealand Pharma's petrelintide shows double-digit weight loss with remarkably few side effects, positioning it to challenge GLP-1 giants in the $100B market.

DBV Technologies heads to a key investor conference with its VIASKIN patch for peanut allergies. Can its science and strategy convince Wall Street?

BioCardia’s innovative cell therapy for heart failure receives a crucial FDA signal, potentially paving the way for a new treatment paradigm for millions.

As Royalty Pharma prepares for a key investor conference, we explore the powerful, low-profile business model that funds blockbuster drugs and shapes innovation.

A pharma revolution is brewing for Generalized Anxiety Disorder. As prevalence soars, novel therapies targeting new neural pathways promise to upend the market.

The market for T-cell malignancy treatments is set to surpass $1.8B, fueled by a pipeline of innovative drugs aiming to meet vast unmet patient needs.

As Prime Medicine prepares for a key investor conference, its next-gen 'search and replace' gene editing technology faces a crucial test. Can it deliver on its promise?

A call for research on vaccine side effects exposes a deeper crisis: the collapse of public trust in science. Can rigorous data heal a polarized nation?