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RSS FeedPosted by Michael Wonder on 23 Nov 2023
Praxis Precision Medicines receives PRIME designation from the EMA for elsunersen (PRAX-222) for treatment of SCN2A gain of function developmental epilepsies
16 November 2023 - IRE) -- Praxis Precision Medicines today announced that the EMA has awarded its Priority Medicines (PRIME) designation ...
Posted by Michael Wonder on 14 Nov 2023
Atsena Therapeutics receives FDA regenerative medicine advanced therapy designation for ATSN-101 gene therapy for GUCY2D associated Leber congenital amaurosis
14 November 2023 - ATSN-101 has demonstrated clinically meaningful improvements in vision at the highest dose with no drug-related serious ...
Posted by Michael Wonder on 01 Nov 2023
X4 Pharmaceuticals announces FDA acceptance with priority review of US NDA for mavorixafor in WHIM syndrome
31 October 2023 - FDA sets a PDUFA target action date of 30 April 2024. ...
Posted by Michael Wonder on 31 Oct 2023
US FDA awards both rare paediatric disease and orphan drug designations to HG204, a CRISPR RNA editing therapy, for the treatment of MECP2 duplication syndrome
31 October 2023 - HG204 is the world first CRISPR RNA-editing therapy for the treatment of MECP2 duplication syndrome granted ...
Posted by Michael Wonder on 30 Oct 2023
bluebird bio enters into advance agreement to sell priority review voucher, if granted, for $103 million
30 October 2023 - bluebird bio today announced that it has entered into an agreement to sell a rare paediatric disease ...
Posted by Michael Wonder on 26 Oct 2023
Valneva submits Chikungunya vaccine marketing application to EMA and announces CHMP accelerated assessment
25 October 2023 - Valneva today announces the submission of a marketing application with the EMA for approval of the ...
Posted by Michael Wonder on 25 Oct 2023
Annexon receives PRIME designation from the EMA for ANX007 for the treatment of geographic atrophy
24 October 2023 - PRIME designation granted based on Phase 2 ARCHER trial results, which showed meaningful preservation of visual function ...
Posted by Michael Wonder on 24 Oct 2023
Immatics receives FDA regenerative medicine advanced therapy designation for ACTengine IMA203 TCR-T monotherapy
24 October 2023 - Regulatory activities underway with an initial focus on a registration directed trial in melanoma as step ...
Posted by Michael Wonder on 24 Oct 2023
4DMT receives EMA Priority Medicines (PRIME) designation for 4D-150 clinical stage genetic medicine for treatment of wet AMD
23 October 2023 - 4D-150 combines a novel, targeted intravitreal next generation AAV vector with a dual transgene that inhibits four ...
Posted by Michael Wonder on 17 Oct 2023
NorthSea Therapeutics receives FDA rare paediatric disease designation for SEFA-6179 for the treatment of intestinal failure-associated liver disease
17 October 2023 - Rare paediatric disease designation underscores critical need for novel therapies to address IFALD. ...
Posted by Michael Wonder on 16 Oct 2023
Editas Medicine granted FDA regenerative medicine advanced therapy designation for EDIT-301 for the treatment of severe sickle cell disease
16 October 2023 - Editas Medicine today announced that the US FDA granted regenerative medicine advanced therapy designation to EDIT-301, an ...
Posted by Michael Wonder on 16 Oct 2023
Tagrisso plus chemotherapy granted priority review in the US for patients with EGFR mutated advanced lung cancer
16 October 2023 - Decision based on FLAURA2 Phase 3 trial results which extended median progression-free survival by nearly 9 ...
Posted by Michael Wonder on 14 Oct 2023
Intellia Therapeutics receives Priority Medicines (PRIME) designation from the EMA for NTLA-2002, an investigational in vivo CRISPR genome editing treatment for hereditary angioedema
13 October 2023 - Intellia Therapeutics today announced that the EMA has granted Priority Medicine (PRIME) designation to NTLA-2002 for ...
Posted by Michael Wonder on 07 Oct 2023
Zatolmilast, an investigational treatment for Fragile X syndrome, receives rare paediatric disease designation from the US FDA
27 September 2023 - Shionogi announced the US FDA has granted Tetra Therapeutics, a Shionogi Group Company, rare paediatric disease designation ...
Posted by Michael Wonder on 03 Oct 2023
Rocket Pharmaceuticals announces FDA acceptance of biologics license application with priority review for RP-L201 (marnetegragene autotemcel) for the treatment of severe leukocyte adhesion deficiency-I
2 October 2023 - PDUFA target action date is 31 March 2024. ...