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RSS FeedPosted by Michael Wonder on 23 Apr 2026
FDA approves first ever gene therapy for treatment of genetic hearing loss under National Priority Voucher Program
23 April 2026 - Groundbreaking AAV-based gene therapy offers potential treatment for patients with OTOF gene-associated severe-to-profound and profound hearing loss. ...
Posted by Michael Wonder on 23 Apr 2026
Otarmeni (lunsotogene parvec-cwha) approved by FDA as first and only gene therapy for genetic hearing loss; Regeneron to provide Otarmeni for free in the US
23 April 2026 - Regeneron Pharmaceuticals today announced the US FDA has granted accelerated approval for Otarmeni (lunsotogene parvec-cwha), the first ...
Posted by Michael Wonder on 17 Apr 2026
Medera receives FDA fast track designation for gene therapy targeting Duchenne muscular dystrophy associated cardiomyopathy
16 April 2026 - Medera today announced that the US FDA has granted fast track designation to AAV-SERCA2a, an investigational ...
Posted by Michael Wonder on 15 Apr 2026
US FDA grants RMAT designation to GS-100, Grace Science’s gene therapy to treat NGLY1 deficiency
14 April 2026 - Grace Science announced today that the US FDA has granted regenerative medicine advanced therapy designation to GS-100 ...
Posted by Michael Wonder on 10 Apr 2026
Replimune receives complete response letter from the FDA for RP1 biologics license application for the treatment of advanced melanoma
10 April 2026 - Replimune Group today announced that the company received a complete response letter from the US FDA ...
Posted by Michael Wonder on 08 Apr 2026
Ray Therapeutics receives FDA regenerative medicine advanced therapy designation for RTx-015 in retinitis pigmentosa
1 April 2026 - Ray Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to its ...
Posted by Michael Wonder on 02 Apr 2026
Ultragenyx announces US FDA acceptance of BLA resubmission for UX111 AAV gene therapy to treat Sanfilippo syndrome type A
2 April 2026 - Ultragenyx Pharmaceutical today announced the US FDA has accepted for review the resubmitted biologics license application seeking ...
Posted by Michael Wonder on 31 Mar 2026
FDA approves new high dose regimen of Spinraza (nusinersen) for spinal muscular atrophy
30 March 2026 - The approval was anchored on data from the pivotal DEVOTE study that investigated the efficacy and safety ...
Posted by Michael Wonder on 27 Mar 2026
MeiraGTx announces FDA breakthrough therapy designation for AAV2-hAQP1 for the treatment of grade 2 and grade 3 radiation-induced xerostomia
26 March 2026 - FDA granted breakthrough therapy designation for AAV2-hAQP1 for the treatment of Grade 2 and Grade 3 late ...
Posted by Michael Wonder on 27 Mar 2026
FDA approves first gene therapy for severe leukocyte adhesion deficiency type I
26 March 2026 - The US FDA today approved Kresladi (marnetegragene autotemcel), the first gene therapy for the treatment of severe ...
Posted by Michael Wonder on 23 Mar 2026
Ionis announces zilganersen new drug application for Alexander disease accepted by FDA for priority review
23 March 2026 - Ionis Pharmaceuticals today announced that the US FDA has accepted for priority review the new drug application ...
Posted by Michael Wonder on 10 Mar 2026
Sangamo Therapeutics advances rolling submission of BLA to US FDA for ST-920 in Fabry disease
9 March 2026 - Sangamo Therapeutics today announced advancement of the rolling submission of a BLA to the FDA seeking ...
Posted by Michael Wonder on 09 Mar 2026
Precision BioSciences receives FDA fast track sesignation for PBGENE-DMD
9 March 2026 - Precision BioSciences today announced that the US FDA has granted fast track designation to PBGENE-DMD for the ...
Posted by Michael Wonder on 24 Feb 2026
Ultragenyx announces US FDA acceptance and priority review of the biologics license application for DTX401 AAV gene therapy for glycogen storage disease type Ia
23 February 2026 - Ultragenyx today announced the US FDA has accepted for review the biologics license application seeking approval of ...
Posted by Michael Wonder on 23 Feb 2026
Tessera Therapeutics receives US FDA fast track and orphan drug designations for its lead in vivo gene editing program TSRA-196 for the treatment of adults with AATD
23 February 2026 - Tessera Therapeutics today announced that the US FDA has granted fast track and orphan drug designations to ...