Mean ABR: FDA label calculated the mean reduction as 52% as compared to 80% reported by $BMRN FDA label more onerous than EMA label for Roctavian or FDA label for Hemegenix #liver #monitoring requirements WILL #EXCLUDE ~85% OF ALL US HEMA PATIENTS

Today we report in Nature Biomedical Engineering the systematic application of six recent prime editing developments to enable efficient and functional correction of CFTR F508del, the predominant mutation that causes cystic fibrosis (CF), in primary airway cells from CF patients.

$FDMT $ADVM honestly I didn't know people were still interested in gene therapy for wAMD concerns about steroid use, durability, inflammation, etc pretty strong believer that the best targets for gene therapy are for fatal genetic diseases with limited or no alternative

Today we are thrilled to share positive topline results from the pivotal Phase 3 AFFINE trial, evaluating the gene therapy candidate we are co-developing with and licensing to @Pfizer for the treatment of adults w/moderately severe to severe #hemophiliaA. bit.ly/3YgTRUV

What happened? -- Mother of young adult w. Duchenne, stood up at Parent Project Muscular Dysytrophy (PPMD) conference, criticized Sarepta $SRPT exec on stage during Q&A gene therapy session. Elevidys is the "Betamax of gene therapy," she said, faults Sarepta for withholding key

$SRPT surprised to learn from Roche earnings that DMD gene therapy Elevidys approved in 6 ex-US countries with sales of $32M.

$SRPT Posting for the benefit of those not following STAT+ or following Sheep of Wall Street ...everyone should be able to see this

#RNAediting Co Airna raises another $60M in Series A (led by German VC Forbion) to advance what they hope to be best-in-class A-->I AATD program. Investor appear to be bullish ahead of $WVE clinical readout which will be extremely important for field. $PRQR $BEAM

Gene therapy landscape Pulled together 42 companies and charted the preclinical and clinical assets of each, segmented by approach and vector Some takeaways:

RNA- & Epigenome-editing have the benefit to come without the long-term commitment of genome editing or gene addition (ie AAV based gene therapy)

Published today in nature, we describe an approach for single-molecule protein reading on Oxford Nanopore arrays. By utilizing ClpX unfoldase to ratchet proteins through a CsgG nanopore, we achieved single-amino-acid sensitivity. nature.com/articles/s4158…

Potent and selective repression of SCN9A by engineered zinc finger repressors for the treatment of neuropathic pain biorxiv.org/cgi/content/sh… #biorxiv_neursci

Cool

$SGMO preprint

Will last years stellar FLT201 data in Gaucher disease hold up? #AAV

Very relevant to #AAV #genetherapy since anti-AAV antibodies are preventing a a lot of patients to get treatment or participate in clinical trials. For #Beqvez it was published that 60% of screened patients were ineligible due to anti-AAV antibodies

Immune system one pager

$WVE $PRQR significant

Explaining the problematic ‘yo-yo’ effect often seen with dieting. 🎂 🍰👇 Adipose tissue retains an epigenetic memory of obesity after weight loss | Nature nature.com/articles/s4158…

Bye twitter.