The BioPharm Brief: CAR-T, RNAi, and Inhaled ASOs

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Here’s what we’re talking about today.

BrainChild Bio has raised $116 million and launched a pivotal phase 2 trial of its CAR-T therapy for diffuse intrinsic pontine glioma, or DIPG. The trial is evaluating BCB-276, a B7-H3-targeted autologous CAR-T, in children and young adults with newly diagnosed DIPG following radiation. The therapy is delivered directly into the cerebrospinal fluid, aiming to overcome some of the challenges posed by the blood-brain barrier. Earlier Phase 1 data reported a median overall survival of 19.8 months from diagnosis, although those findings came from a small, single-center, nonrandomized study.

In obesity, Moonwalk Biosciences has raised $70 million to advance its adipose-targeted RNAi program. The company’s lead candidate, MW101, is designed to deliver siRNA directly to adipose tissue and target non-incretin pathways involved in energy metabolism, adipogenesis, lipolysis, and thermogenesis. Preclinical studies showed reductions in body weight and fat mass while preserving lean mass, with first-in-human testing planned for late 2027.

And Aptar Pharma is partnering with Aceso Therapeutics to advance an inhaled antisense oligonucleotide for cystic fibrosis. Aptar’s Nanopharm will lead formulation development and device assessment for ACT-101, which is designed to address CFTR pre-mRNA processing in patients with the F508del mutation. The collaboration highlights the formulation and delivery challenges that come with bringing nucleic acid therapies directly to the lungs.

That’s The BioPharm Brief for today. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways:

• BrainChild Bio raised $116 million and launched a pivotal Phase 2 trial of BCB-276, a B7-H3-targeted CAR-T therapy for newly diagnosed DIPG.
• Moonwalk Biosciences raised $70 million to advance MW101, an adipose-targeted siRNA therapy for obesity, toward first-in-human testing.
• Aptar Pharma and Aceso Therapeutics partnered to advance ACT-101, an inhaled antisense oligonucleotide designed to address CFTR dysfunction in cystic fibrosis.