The Connector's Corner — Week of August 21, 2026

Your weekly connection to biotech, rare disease talent, and patient-centric innovation — from Ronith Dunn and BlueGene Partners.

A Note From Me

Happy Friday, connectors.

August has been anything but a slow summer month. In the last two weeks alone, the FDA has approved a first-of-its-kind treatment for FOP, a gene therapy for Von Gierke disease, and a new option for recurrent respiratory papillomatosis — while the MS/Neuro space quietly notched two of the more interesting regulatory milestones of the year. On the talent side, three of the largest rare-disease and neuroscience shops in the market — Sarepta, BioNTech, and Juvena — all announced new CEOs. When leadership shifts at that scale, the ripple effects on hiring, pipeline priorities, and advocacy partnerships are real.

A few updates from BlueGene Partners: we're actively running searches for Hansa Biopharma (US Policy & Patient Advocacy; US Trade & Distribution), Liquidia (NYC Territory Account Manager supporting the PAH launch), and are finalizing an MSA to embed with Savara on fractional recruiting. If you're an operator with rare-disease commercial, market access, or advocacy DNA — or know someone who is — reply to this email. I read every note.

A new connection I have to tell you about. This week I had the honor of meeting Luke Rosen — rare disease dad, KIF1A.ORG co-founder, former actor, FDNY firefighter, cancer survivor, and one of the most generous humans I've encountered in this space. Luke's daughter Susannah has KIF1A-associated neurological disorder, and out of that reality he has built a foundation, driven an N-of-1 antisense oligonucleotide program, launched Rescue 7 (firefighters for patients), and testified in a Congressional briefing on rare precision medicine alongside biotech leaders. I am lucky to be in a space where people like Luke exist. A few of the pieces he shared with me that I think this whole community should read are in "Voices from the Community" below.

❝

"So my answer to your question is that it's all about the elevator, I guess. Who is there when it opens on the hardest day of your life, and who takes it down with you a few hours later is what matters."

— Luke Rosen, "The Elevator" (KIF1A.ORG)

Grateful you're here.

— Ronith

Industry Pulse

Kyverna's miv-cel earns RMAT for non-active secondary progressive MS. The FDA granted regenerative medicine advanced therapy designation to miv-cel for naSPMS based on investigator-initiated data out of Stanford and UCSF — a meaningful signal for CAR-T in progressive MS (Kyverna via Business Insider).

Quantum BioPharma's Lucid-MS clears FDA to enter Phase 2 in progressive MS. The IND clearance on Aug. 10 lets Quantum begin dosing patients with a first-in-class myelin-protecting agent that works without immunosuppression — a differentiated mechanism in a space dominated by B-cell depleters (Quantum BioPharma press release).

Regeneron's Pasatru wins first FDA approval in fibrodysplasia ossificans progressiva. The Aug. 19 nod puts Regeneron in direct competition with Ipsen's Sohonos in an ultra-rare bone disorder — a category where advocacy relationships often determine commercial trajectory (Reuters via Yahoo Finance).

Ultragenyx's Genglycos becomes first approved gene therapy for Von Gierke disease. The Aug. 19 approval for glycogen storage disease type Ia adds another chapter to Ultragenyx's rare metabolic playbook (Reuters).

A big month for rare disease at the FDA. August delivered four historic rare-disease approvals (Jazz's Modeyso, Insmed's Brinsupri, Precigen's Papzimeos, and Regeneron's Pasatru) — offset by a Complete Response Letter for PTC Therapeutics' vatiquinone in Friedreich's ataxia (BioSpace).

Patient & Advocacy Watch

A first-ever clinical trial framework for Charcot-Marie-Tooth disease. CMT advocacy organizations, clinicians, and industry aligned on shared recommendations published in the Journal of the Peripheral Nervous System — including flexible trial designs to limit placebo exposure and enrollment based on clinical phenotype rather than genetic subtype. This is the kind of pre-competitive advocacy work that materially speeds enrollment across rare neuromuscular programs (MDA).

Cedars-Sinai, UCSF, and Cambridge identify at-risk brain cells in MS. New multicenter research pinpoints gray-matter neurons especially prone to DNA damage during MS inflammation — a finding that could shape how progressive MS is measured and treated (Cedars-Sinai).

A low-cost MS option gets validated head-to-head. A Norwegian-Swedish trial in NEJM showed rituximab is as effective as ocrelizumab in newly diagnosed relapsing MS — a result with meaningful access implications for patients and payers globally (Medical Xpress).

Talent Angle — Leadership Moves to Watch

Sarepta names Michael Severino CEO. The former Tessera CEO and longtime AbbVie president takes the helm of the rare-disease drugmaker at a critical inflection point post-Ingram. Expect leadership team refreshes, especially in medical affairs and patient advocacy (STAT News).

Juvena Therapeutics appoints Julie Anne Smith CEO. A seasoned rare-disease and neuromuscular exec joins as Juvena advances JUV-161 into Phase 2 and executes its Eli Lilly collaboration — one to watch for build-out hiring (BioSpace).

BioNTech taps Sobi's Guido Oelkers as next CEO. Oelkers, who quadrupled revenues at rare-disease specialist Sobi, will start by Feb. 1, 2027 as BioNTech accelerates its oncology pivot — the Sobi succession itself will trigger movement across European rare-disease leadership (STAT News).

Neurvati Neurosciences deepens its bench. J. Michael Ryan, M.D. joins as CMO with Anne-Marie Li-Kwai-Cheung promoted to Chief Development Officer and Elliott Ruiz to CFO — a signal that GRIN Therapeutics is entering its next growth phase (BioSpace).

Kenai Therapeutics adds Mark Gergen to its board. The neurology-focused clinical-stage biotech brings on a 30-year life-sciences operator/dealmaker (Kenai).

BlueGene Partners is actively searching for:

Head of US Policy & Patient Advocacy — Hansa Biopharma
US Trade & Distribution Lead — Hansa Biopharma
Territory Account Manager, NYC (PAH launch) — Liquidia

If any of these are on your radar, or if you know a rare-disease commercial or advocacy leader considering their next move, hit reply.

Voices from the Community — Luke Rosen's Reading List

A curated set of pieces Luke shared with me this week. Every one is worth your time:

The Elevator — Luke's essay on getting a rare-disease diagnosis, republished in NYT Magazine. If you read only one, read this.
The Hope of an N:1 Trial: Antisense Makes Sense — inside the community effort behind an individualized ASO program.
Enterprises of Great Pitch and Moment: Biotech Pitch / Advocacy ROI — required reading for founders on why patient advocacy is not a soft skill.
Cost Analysis of Starting a Rare Disease Foundation — the real numbers, from someone who's done it.
Why We Need a Mouse — on preclinical models and the human cost of not building them.
Unlocking sociocultural and community factors for the global adoption of genomic medicine — peer-reviewed work co-authored by Luke on equitable rare-disease access.
The Moth / Story Collider: A New and Incredibly Unthinkable Normal — Luke telling his family's story live.
Luke on CNBC and on Fox — for how he moves the conversation into mainstream media.

Companies to Follow This Week

Kyverna Therapeutics — CAR-T in progressive MS just became a real conversation
Quantum BioPharma — first-in-class myelin protection heading into Phase 2
Juvena Therapeutics — new CEO, Lilly deal, Phase 2 all in one quarter
Neurvati / GRIN Therapeutics — leadership build-out signals scale-up
Capricor Therapeutics — deramiocel PDUFA (Aug. 22) is the next binary event in DMD

What I'm Grateful For

This week I'm grateful for the CMT community — patient orgs, clinicians, and industry — for showing what pre-competitive collaboration actually looks like. Building a shared trial framework in a rare disease is slow, unglamorous, generous work. It's exactly the kind of patient-centric innovation this newsletter exists to celebrate.

And I'm grateful for Luke Rosen — for the generosity of his time, the depth of his story, and the reminder that the people who move rare disease forward are usually the people who had no choice but to. Meeting connectors like Luke is why I do this.

And I'm grateful for you — for reading, replying, forwarding, and connecting. Every intro you make expands what's possible for the people we serve.

Until next Friday,

Ronith Dunn
Founder & Chief Connector, BlueGene Partners
theconnectorscorner.beehiiv.com