Last Week in Biomedicine | June 14–20 | Part 1 of 2: CRISPR HAE Cure, Narcolepsy Orexin Breakthrough

Last Week in Biomedicine | June 14–20 | Part 1 of 2: CRISPR HAE Cure, Narcolepsy Orexin Breakthrough

This week opened with one of the most significant gene-editing milestones in clinical medicine: a single CRISPR infusion cut hereditary angioedema attacks by 87 percent in a Phase 3 trial, published the same day in the New England Journal of Medicine. Plus a first-in-class drug that may reshape how we treat narcolepsy, and the first new oral antifungal class in over 20 years.

In Part 1 we cover:

1. Intellia Therapeutics — Lonvoguran ziclumeran (lonvo-z) for hereditary angioedema (Phase 3)

2. Takeda Pharmaceutical — Oveporexton (TAK-861) for narcolepsy type 1 (Phase 3)

3. F2G / Shionogi — Olorofim for invasive aspergillosis (Phase 3)


The Intellia readout is the headline of the week. Hereditary angioedema causes sudden, painful and sometimes life-threatening swelling attacks, and current treatments require regular injections to suppress them. Lonvo-z is given once — a tiny package of CRISPR machinery shuts down the gene responsible for the attacks inside liver cells permanently — and 81 percent of patients were completely attack-free at one year. If this holds up, an entire category of chronic disease management could be replaced by single-dose curative gene

editing.


Part 2 drops Thursday morning. Subscribe at TrialBreaks.com.


#biomedicine #clinicaltrials #science #CRISPR #genetherapy #raredisease

#neurology #narcolepsy #orexin #infectiousdisease #antifungal #immunology #HAE

#aspergillosis #drugdevelopment


Tämä jakso on lisätty Podme-palveluun avoimen RSS-syötteen kautta eikä se ole Podmen omaa tuotantoa. Siksi jakso saattaa sisältää mainontaa.

Jaksot(17)

Last Week in Biomedicine | Jun 29–Jul 3 | Part 2 of 2: Baby Pompe Disease Trial, DLBCL Combo Win

Last Week in Biomedicine | Jun 29–Jul 3 | Part 2 of 2: Baby Pompe Disease Trial, DLBCL Combo Win

Part 2 covers three more standout stories from the same week: a Phase 3 trial that gives babies with a devastating enzyme disease a fighting chance, a first-of-its-kind cell therapy approved to make b...

9 Heinä 36min

Last Week in Biomedicine | Jun 29–Jul 3 | Part 1 of 2: CRISPR for Toddlers, KRAS Lung Cancer Win

Last Week in Biomedicine | Jun 29–Jul 3 | Part 1 of 2: CRISPR for Toddlers, KRAS Lung Cancer Win

CRISPR gene editing crossed a major line this week: the FDA now allows Casgevy — the first CRISPR therapy of any kind — for children as young as 2 with sickle cell disease, meaning we can potentially ...

7 Heinä 43min

Last Week in Biomedicine | June 22–26 | Part 2 of 2: Thyroid Eye Disease, ADHD + Anxiety, Skyrizi Kids

Last Week in Biomedicine | June 22–26 | Part 2 of 2: Thyroid Eye Disease, ADHD + Anxiety, Skyrizi Kids

Part 2 covers a new drug for the bulging-eye autoimmune disease, the first Phase 3 ADHD trial designed for patients who also have anxiety, and a major pediatric expansion for a top-selling psoriasis b...

2 Heinä 41min

Last Week in Biomedicine | June 22–26 | Part 1 of 2: LSD Depression Phase 3, Trodelvy TNBC Approval

Last Week in Biomedicine | June 22–26 | Part 1 of 2: LSD Depression Phase 3, Trodelvy TNBC Approval

This week, an LSD-based therapy hit its main goal in a Phase 3 depression trial — and two huge breast cancer approvals landed at the FDA on the same week. In Part 1 we cover:1. Definium Therapeutics —...

30 Kesä 36min

Last Week in Biomedicine | June 14–20 | Part 2 of 2: Mezigdomide Myeloma, HCM Heart Drug

Last Week in Biomedicine | June 14–20 | Part 2 of 2: Mezigdomide Myeloma, HCM Heart Drug

Part 2 of our week of June 14–20, 2026 wrap-up covers a new molecular glue for relapsed multiple myeloma that cut disease progression by half, a heart drug that may finally work forboth forms of hyper...

25 Kesä 25min

Last Week in Biomedicine | June 6–13 | Part 2 of 2: CagriSema Diabetes, Sickle Cell Plenary

Last Week in Biomedicine | June 6–13 | Part 2 of 2: CagriSema Diabetes, Sickle Cell Plenary

Part 2 of our June 6–13 wrap-up: Novo Nordisk's CagriSema beats semaglutide alone in three Phase 3 diabetes trials, Takeda's zasocitinib pill outperforms Sotyktu head-to-head in psoriasis, and Agios's...

18 Kesä 21min

Last Week in Biomedicine | June 6–13 | Part 1 of 2: Retatrutide Weight Loss, Weekly HIV Pill

Last Week in Biomedicine | June 6–13 | Part 1 of 2: Retatrutide Weight Loss, Weekly HIV Pill

A blockbuster week in biomedicine — Eli Lilly's retatrutide produced weight loss approaching bariatric surgery in Phase 3, and Gilead and Merck's once-weekly oral HIV pill cleared two Phase 3 trials. ...

16 Kesä 20min

Suosittua kategoriassa Terveys ja hyvinvointi

unicast
tiedenaiset-podcast
vakeva-elama-viisaampi-mieli-vahvempi-keho
rss-pitaisko-erota
psykopodiaa-podcast
meditaatiot-suomeksi
voi-hyvin-meditaatiot-2
katilon-kahvitunti
rss-uplevel-by-sonja-hannus
rss-vapaudu-voimaasi
rss-pt-paahtio
rss-kuumilla-aalloilla
terapiassa
junnut-pelissa
selviytyjat-tarinoita-elamasta
docemilia
paritellen
kehorauhaa
rakkaudella-karita
rss-sanelunpurku-podcast