Gene therapy shows sustained benefit in Wiskott–Aldrich syndrome
1. In this analysis of data from over two dozen individuals with Wiskott-Aldrich syndrome, treatment with etuvetidigene autotemcel (etu-cel) was ...
Tag: gene therapy
![2 Minute Medicine: Pharma Roundup: Price Hikes, Breakthrough Approvals, Legal Showdowns, Biotech Expansion, and Europe’s Pricing Debate [May 12nd, 2025]](https://www.2minutemedicine.com/wp-content/uploads/2025/05/ChatGPT-Image-May-12-2025-at-10_22_23-AM-350x250.png)
2 Minute Medicine: Pharma Roundup – New oral gonorrhea antibiotic Nuzolvence (zoliflodacin), late‑December cardiovascular and oncology drug approvals, first targeted complement inhibitor therapy for HSCT‑TMA (Yartemlea), and antibody‑drug conjugate (ADC) safety concerns with ifinatamab deruxtecan (I‑DXd) [January 2026]
Oral gonorrhea antibiotic Nuzolvence approved just weeks ago The Food and Drug Administration recently approved Nuzolvence (zoliflodacin) as a first‑in‑class ...
2 Minute Medicine: Pharma Roundup – Breakthrough ALS gene therapy, new adult pneumonia vaccine, needle-free obesity treatment, and Pfizer’s business overhaul [October 28, 2025]
Amryta’s Sodesta gene therapy gains accelerated approval for ALS The Food and Drug Administration has granted accelerated approval to Sodesta, ...
AAV gene therapy shows durable benefit in hemophilia B
1. In this prospective cohort study, a single intravenous dose of scAAV2/8-LP1-hFIXco gene therapy in men with severe hemophilia B ...
AAV9.LAMP2B gene therapy shows clinical benefit in Danon disease
1. In seven patients with Danon disease, a single infusion of adeno-associated virus serotype 9 carrying transgene LAMP2B (AVV9.LAMP2B) therapy ...
#VisualAbstract: Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B is Superior to Prophylaxis
Click here to read this study in NEJM. ©2024 2 Minute Medicine, Inc. All rights reserved. No works may be reproduced ...
Taysha Gene Therapies: TSHA-102 Gene Therapy Improves Quality of Life in Patients with Rett Syndrome
A patient who received TSHA-102 had durable improvements in motor skills, communication, and socialization. TSHA-102 was well tolerated with no ...
Intrathecal gene therapy as a treatment for giant axonal neuropathy
1. In this dose-escalation study, using intrathecal gene transfer with scAAV9/JeT-GAN demonstrated potential improvement in motor function. 2. Adverse events ...
Pfizer: Beqvez Gene Therapy Decreases Bleeding Rates in Hemophilia B Patients
Hemophilia B patients given Beqvez had a decreased incidence of total bleeding Beqvez reduced the use of routine factor IX ...
Gene therapy cost-effective for treating sickle cell disease in simulation models
1. In this cost analysis, compared to standard care, it was found that gene therapy for sickle cell disease (SCD) ...
