Blog
RSS FeedPosted by Michael Wonder on 22 May 2026
Otarmeni (lunsotogene parvec) receives EMA filing acceptance for genetic hearing loss
22 May 2026 - Regeneron Pharmaceuticals today announced the EMA has accepted for review under accelerated assessment the marketing authorisation application ...
Posted by Michael Wonder on 18 May 2026
Qalsody: new data has allowed for a reassessment by the HAS
21 April 2026 - The drug Qalsody (tofersen) aims to slow the progression of a rare genetic form of ALS (amyotrophic ...
Posted by Michael Wonder on 17 May 2026
MHRA approves beremagene geperpavec (Vyjuvek) for the treatment of dystrophic epidermolysis bullosa
15 May 2026 - The MHRA has today approved beremagene geperpavec (Vyjuvek) for the treatment of wounds in patients with dystrophic ...
Posted by Michael Wonder on 15 May 2026
Routine NHS access to spinal muscular atrophy treatments offers new hope for patients and families
14 May 2026 - People living with spinal muscular atrophy in England are set to gain routine access to two potentially ...
Posted by Michael Wonder on 01 May 2026
Ray Therapeutics granted Priority Medicines (PRIME) designation from the EMA for RTx-015 in retinitis pigmentosa
29 April 2026 - PRIME designation follows recent FDA regenerative medicine advanced therapy designation, continues regulatory momentum for RTx-015. ...
Posted by Michael Wonder on 29 Apr 2026
Intellia Therapeutics initiates rolling submission of biologics license application to FDA for lonvoguran ziclumeran as a one time treatment for hereditary angioedema
27 April 2026 - Expect to complete biologics license application submission in second half of 2026; anticipate launch in first ...
Posted by Michael Wonder on 27 Apr 2026
Calls for $1 million gel to be available in Oz to fight rare genetic disease
26 April 2026 - A little boy suffering a rare disease that leaves the skin as delicate as butterfly wings ...
Posted 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 ...