Gene Therapies

An N-of-3 pilot study reveals how Krystal Biotech's new intratumoral immunotherapy could replace lifelong surgeries with systemic immune clearance.

By targeting the genetic roots of BAG3 cardiomyopathy with a novel low-dose capsid, Affinia aims to solve gene therapy’s systemic toxicity bottleneck.

By bypassing the blood-brain barrier, AviadoBio's novel gene therapy halts key neurodegeneration markers in a devastating form of genetic dementia.

A breakthrough gene therapy restores natural hearing in children, challenging mechanical implants and reshaping the economics of genomic medicine.

As the Progeria Research Foundation honors pioneer David R. Liu and philanthropists Cathy and Dave Gravel, the ultimate biological network upgrade approaches clinical reality.

The 2026 Nobel Prize in Medicine honors three scientists who turned light into a neurological remote control, paving the way for revolutionary clinical therapies.

Coave Therapeutics is leveraging AI and novel suprachoroidal delivery to disrupt the wet AMD market, targeting the end of chronic eye injections.

Ajinomoto Bio-Pharma Services' latest rebrand signals a vital shift, prioritizing deep technical mastery in complex therapies over the one-stop-shop model.

Biotech challengers are leveraging gene therapy and oral molecules to dismantle the legacy infusion model in the $1.7 billion Fabry disease market.

Taysha Gene Therapies leverages novel auto-regulatory technology and a strategic regulatory playbook to pioneer a breakthrough gene therapy for Rett syndrome.

A new partnership between the TECPR2 Research Foundation and CDMO Forge Biologics highlights a shift: families are now acting as sophisticated biotech operators.

Following its formation via private equity, Minaris reorganizes to tackle the viral vector capacity crunch and navigate the post-BIOSECURE Act landscape.

A massive €40 million seed round for an Italian biotech signals a paradigm shift in treating intractable genetic disorders like Fragile X Syndrome.

The strategic alliance between ProBio and Kun Tuo signals a shift in how global biopharma navigates the fragmented cell and gene therapy clinical landscape.

IASO208’s FDA clearance marks a critical leap for in vivo CAR-T therapy, promising to strip away manufacturing bottlenecks and reshape global biopharma.

A novel non-viral gene therapy shows early promise in treating Geographic Atrophy, targeting the root cause of iron overload to potentially reverse vision loss.

A landmark TGA license for Australia's first viral vector CDMO signals a strategic shift, promising to de-risk supply chains and accelerate advanced therapies.

With new backing from specialist fund SCI Ventures, ViaNautis is deploying its novel gene delivery platform to develop regenerative therapies for paralysis.

Encoded Therapeutics' ETX101 shows potential to not only halt seizures but also reverse developmental delays, suggesting a paradigm shift for rare neurological disorders.

As Neurogene prepares for a key investor conference, all eyes are on its lead candidate for Rett syndrome. The company bets its proprietary EXACT™ technology can solve a critical gene therapy challenge, holding immense promise for patients and investors alike.