Rare Disease & Gene Therapy — July 06, 2026
| BridgeBio’s Attruby posted kidney-protective signals in ATTR-CM that Jefferies calls “distinct” from Pfizer’s tafamidis. Scribe Therapeutics filed to take its CasX gene-editing platform public and non-genetic approaches advanced in ultra-rare CNS disease.
Today’s top developments:
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What to Watch
- Wainua Phase 3 ATTR-CM readout — AstraZeneca and Ionis Phase 3 ATTR-CM data for Wainua (eplontersen), expected late August 2026, will test how BridgeBio’s Attruby cardiorenal differentiation holds up against an antisense competitor entering the cardiomyopathy indication.
- Scribe IPO prospectus — Scribe Therapeutics’ filing is the key document to watch for disclosure of its lead rare disease indication, clinical timeline, and cash runway assumptions underpinning the offering valuation.
- PWS microbiome trial — recruitment completion and interim readout timing from the Fudan prebiotic/probiotic study will clarify whether gut microbiome modulation produces measurable hyperphagia or body-composition signals in pediatric patients.
- NHGRI natural history data — analytics outputs could inform FDA discussions on single-arm trial acceptability for rare genetic disease gene therapy programs seeking accelerated approval pathways.
This brief highlights the edition’s top stories. Read the full July 06, 2026 edition → for all stories and analysis — or browse the Rare Disease & Gene Therapy archive.