Korro Bio, Inc.: KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on ...
Korro Bio gets Orphan Drug Designation from FDA for its genetic disorder treatment
Korro Bio received U.S. FDA's orphan drug designation for investigational medicine KRRO-110 to treat Alpha-1 Antitrypsin ...
Korro Bio's KRRO-110 Gets FDA Orphan Drug Designation
Korro Bio disclosed that the Food and Drug Administration granted orphan drug designation to its potential genetic disorder drug KRRO-110. Shares rose 17.6% to $29 in premarket trading. The stock is ...
KRRO-110 Receives Orphan Drug Designation from U.S. FDA for Alpha-1 Antitrypsin Deficiency
KRRO-110 is the first RNA editing development candidate from Korro’s proprietary OPERA TM platform and is currently being evaluated in the Phase 1/2a REWRITE clinical study for AATD. Dosing of the ...
Korro Bio receives ODD from U.S. FDA for AATD
Korro Bio (KRRO) announced that the U.S. Food and Drug Administration has granted orphan drug designation, ODD, to the investigational medicine ...
The Manila Times1d
Cellectis Reports Financial Results for the Fourth Quarter and Full Year 2024 and Provides a Business Update
In 2025, Cellectis will continue to focus its efforts and expenses on advancing its core clinical trials BALLI-01 and NATHALI ...
TechBullion1d
Disease Modifying in Neurodegenerative Disease Treatment: An Interview with Alon Ben-Noon, CEO & Founder of NeuroSense Therapeutics
Noon, is tackling ALS and other neurological disorders with a bold, multi-targeted approach. Fueled by a personal encounter with an ALS advocate, Ben-Noon assembled a team of top scientists to develop ...
Quoin Pharmaceuticals, Inc.: Quoin Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full-Year 2024 Financial Results
Quoin Pharmaceuticals Ltd. (NASDAQ: QNRX) (the "Company" or "Quoin"), a late clinical-stage specialty pharmaceutical company focused on rare and orphan ...
MindSite News on MSN1d
Opioid Orphans: Grandparents Struggle to Raise Children Left Behind
Despite billions raised from opioid settlements, grandparents raising grandchildren get little. The past five years has seen ...
Healio2d
FDA grants fast track designation to X-linked retinoschisis treatment
The FDA granted fast track designation to ATSN-201 for the treatment of X-linked retinoschisis, according to a press release ...
Fierce Pharma2d
Argenx's Vyvgart tops Trinity’s latest launch success analysis, beating out several Big Pharma debuts
Treatments for autoimmune diseases and those in orphan markets are on the up and up, as are drugmakers’ first launches, ...
Business Wire2d
Geron Announces European Commission Approval of RYTELO® (imetelstat), a First-in-Class Telomerase Inhibitor, for the Treatment of Adults With Transfusion-Dependent Anemia Due ...
Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, today an ...