Daily Snap - 12. September 2025

Author

Joachim Eeckhout
September 12, 2025

 

Good morning! Researchers at Mass General Brigham have developed a custom gene editing technique that corrected the ACTA2 mutation causing multisystemic smooth muscle dysfunction syndrome (MSMDS) in mice. Similar to the personalized CRISPR therapy used earlier this year for infant KJ Muldoon (a first in history), it uses viral vectors to target smooth muscle rather than liver cells. The team, which also contributed to KJ’s treatment, aims to move the therapy into human trials for this deadly, single-mutation genetic disease. Here's some news to cheer you up before the weekend!

Enjoy today’s read!

—Joachim E.

SNIPPETS

What’s happening in biotech today?

💸Phase II fuel: Maze Therapeutics’ stock surged over 54% following positive Phase I data for its oral small molecule candidate MZE782, targeting SLC6A19, which showed significant increases in urinary excretion of phenylalanine and glutamine in healthy volunteers, markers suggesting potential efficacy in treating phenylketonuria (PKU) and chronic kidney disease (CKD). The compound was well-tolerated, with no severe adverse events, and displayed pharmacodynamic effects aligned with potential therapeutic benefit, including a dose-dependent dip in eGFR, a known indicator of long-term kidney protection. To support planned Phase II studies in PKU and CKD, Maze secured $150 million through a private placement involving major healthcare investors.

🏷️Label lift: In recent regulatory developments, the FDA canceled an advisory committee meeting for Travere Therapeutics' Filspari, a potential first-in-class treatment for focal segmental glomerulosclerosis (FSGS), signaling possible confidence in its approval despite earlier trial setbacks. The drug’s application remains under FDA review with a decision expected by January 13, 2026.

⚖️Patent showdown: Arrowhead Pharmaceuticals has filed a lawsuit against Ionis Pharmaceuticals in a Delaware federal court, seeking to invalidate Ionis’ patent No. 9,593,333, which allegedly covers the use of therapies targeting apolipoprotein C-III (APOC3) in treating familial chylomicronemia syndrome (FCS). The legal dispute centers around Arrowhead’s investigational RNAi therapeutic, plozasiran, which aims to reduce APOC3 levels to treat FCS and is currently under FDA review. Ionis’ approved drug Tryngolza, also targeting APOC3, was the first FDA-cleared treatment for FCS. Arrowhead argues its drug is based on independent research, while Ionis asserts its intellectual property rights but denies trying to block plozasiran’s launch.

⛔ Trial halted: Capsida Biotherapeutics has suspended its clinical trial of an experimental gene therapy, CAP-002, after the first child participant died following treatment for STXBP1-related developmental and epileptic encephalopathy, a rare neurodevelopmental disorder. The company disclosed the incident in a letter to the patient community and reported it to the FDA, pledging to investigate the cause and provide a full report. The therapy, which uses a custom-engineered viral vector to deliver a gene to brain neurons, had only recently begun recruiting participants. The child’s death follows other fatalities in gene therapy trials, raising concerns about safety in the field.

💰Gene jackpot: Kriya Therapeutics has raised $320 million in an oversubscribed Series D funding round to advance its pipeline of gene therapies targeting chronic diseases with high unmet medical needs. The funding will support clinical trials, research and development, and the scaling of Kriya’s integrated manufacturing platform. The company focuses on directly administered gene therapies for conditions including thyroid eye disease, geographic atrophy, trigeminal neuralgia, type 1 diabetes, and MASH. This follows a $270 million Series C round raised in 2022.

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