Title: The Connector's Corner — July 9, 2026
Subtitle/preview text (optional): A weekly note from Ronith Dunn
1. A NOTE FROM RONITH
This week I keep coming back to one thought: rare disease keeps quietly winning, even when the headlines are about AI. The FDA just green-lit Casgevy for kids as young as 2 with sickle cell. Ipsen doubled down on rare disease with the Memo Therapeutics deal. And a small gene therapy company most people have never heard of — Neurogene — raised $125M to keep pushing on Rett syndrome, a disease that primarily affects little girls. As an MS warrior and someone who has spent 30+ years placing talent in this space, that's the pattern I want more of: real capital and regulatory momentum flowing to the diseases that used to get overlooked. That's the world I want to help build one great hire, one nonprofit conversation, one patient story at a time.
2. INDUSTRY PULSE
FDA approves Casgevy for children with sickle cell as young as age 2 — the first gene therapy for that age group, granted in just 53 days under the National Priority Voucher program. https://www.globenewswire.com/news-release/2026/07/01/3321045/0/en/FDA-Approves-First-Gene-Therapy-for-Young-Children-with-Sickle-Cell-Disease.html
Ipsen to acquire Memo Therapeutics for up to $796M, adding potravitug (Phase 2 antibody for BK polyomavirus in kidney transplant patients) to its rare disease portfolio. https://www.fiercebiotech.com/biotech/ipsen-gets-memo-penning-eu700m-deal-buy-rare-disease-biotech-clinical-stage-asset
Neurogene prices $125M public offering to advance NGN-401 gene therapy toward a potential approval in Rett syndrome. https://allsci.com/news/funding/public-equity-offerings/neurogene-raises-usd-125m-to-accelerate-rett-syndrome-gene-therapy-toward-approval/
3. PATIENT & ADVOCACY WATCH
Apellis' Empaveli approved by FDA for two rare kidney diseases — the first-ever treatment for primary immune complex membranoproliferative glomerulonephritis, a real win for a long-overlooked patient community. https://www.bioworld.com/articles/722735-apellis-empaveli-approved-in-us-for-two-rare-kidney-diseases
New MS diagnostic guidelines are being rolled out to get patients into treatment sooner after their first symptoms — a meaningful shift for the MS community. https://www.wbrz.com/news/wednesday-s-health-report-ms-diagnostic-guidelines-aim-to-get-patients-into-treatment-sooner
4. NONPROFIT SPOTLIGHT: NORD (National Organization for Rare Disorders)
For 40+ years, NORD has been the connective tissue of the U.S. rare disease community — pushing the Orphan Drug Act forward, running patient assistance programs, and giving 300+ patient organizations a shared voice in Washington. In a week where big-company rare disease deals are making the headlines, it's worth remembering that most of these therapies exist because patient advocates showed up first. If you're a biotech leader reading this, NORD is one of the best doors to walk through if you want to build real patient engagement into your program from day one. https://rarediseases.org/
5. THE TALENT ANGLE
AVEO Oncology announces succession plan: Michael Ferraresso (CCO) elevated to President & CEO effective July 1, and Kevin Peacock named Chief Commercial Officer. https://www.biospace.com/press-releases/aveo-oncology-announces-succession-plan
X4 Pharmaceuticals appoints Kelly Gold (CFO at CAMP4 Therapeutics) to its board. https://markets.businessinsider.com/news/stocks/x4-pharmaceuticals-appoints-kelly-gold-experienced-chief-financial-officer-to-its-board-of-directors-1036289909
Takeda's July 2026 U.S. restructuring takes effect this month under new CEO Julie Kim — 600+ commercial and medical roles displaced. https://intuitionlabs.ai/articles/takeda-restructuring-2026-us-job-cuts-operating-model
6. WHAT I'M READING / WATCHING / GRATEFUL FOR
Reading: "Refuse to Disappear" on Patient Worthy — https://patientworthy.com/collaborative-content-on-patient-worthy/
Grateful for: Every patient advocate who picked up the phone with me this week. You keep me honest, and you keep the mission real.
