Philipp Beck retweeted
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/s41586-0…
$wve OMG, single dose, sustained and substantial #RNAediting in WoMan.
Major, major de-risking of new modality with amazing potential, incl gain of function and protective variants.
$prqr $krro
ir.wavelifesciences.com/news…
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
As I said, protein degraders are cool. Hansa uses a protease from S. pyogenes to rapidly degrade IgG extracellulary. Gen 1 imflimidase EU approved for kidney transplant. Gen 2 HNSA-548 just updated w deep, rapid IgG drop shorturl.at/97TOu
Will last years stellar FLT201 data in Gaucher disease hold up? #AAV
On October 23, we will present new data on FLT201 in #Gaucher disease at the 31st Annual @ESGCT Congress in Rome. During a session on AAV #genetherapy for the treatment of rare diseases, we will share the latest from the Phase 1/2 GALILEO study. bit.ly/3ZJz5y2
$SGMO preprint
Potent and selective repression of SCN9A by engineered zinc finger repressors for the treatment of neuropathic pain biorxiv.org/cgi/content/shor… #biorxiv_neursci
Philipp Beck retweeted
Potent and selective repression of SCN9A by engineered zinc finger repressors for the treatment of neuropathic pain biorxiv.org/cgi/content/shor… #biorxiv_neursci
Cool
Published today in @Nature, we describe an approach for single-molecule protein reading on @nanopore arrays. By utilizing ClpX unfoldase to ratchet proteins through a CsgG nanopore, we achieved single-amino-acid sensitivity. nature.com/articles/s41586-0…
Philipp Beck retweeted
Published today in @Nature, we describe an approach for single-molecule protein reading on @nanopore arrays. By utilizing ClpX unfoldase to ratchet proteins through a CsgG nanopore, we achieved single-amino-acid sensitivity. nature.com/articles/s41586-0…
RNA- & Epigenome-editing have the benefit to come without the long-term commitment of genome editing or gene addition (ie AAV based gene therapy)
Philipp Beck retweeted
Gene therapy landscape
Pulled together 42 companies and charted the preclinical and clinical assets of each, segmented by approach and vector
Some takeaways:
Philipp Beck retweeted
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
Philipp Beck retweeted
#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
airna.com/airna-raises-overs…
Philipp Beck retweeted
$SRPT Posting for the benefit of those not following STAT+ or following @Biohazard3737 ...everyone should be able to see this
Philipp Beck retweeted
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 data.
-- Later, Sarepta demands PPMD edit the video to remove the mother's comments. PPMD agrees. The mother's comments were stricken.
-- PPMD posts the censored video on its web site, then deletes it altogether after the editing incident leaks out.
-- The incident raises questions about the financial ties between Sarepta and PPMD, and whether the drugmaker uses its money to influence, or even muzzle, debate over its Duchenne medicines. It has also bolstered concerns voiced by some parents of young adults with Duchenne that Sarepta is more focused on profiting from its gene therapy, called Elevidys, than providing them with information necessary to prove its efficacy and safety, despite the blessing of the Food and Drug Administration.
-- I spoke to the woman whose comments against Sarepta were removed from the video. I also spoke to two other mothers who have older sons with Duchenne -- all critical of Sarepta for promoting Elevidys for older boys/young adults without adequate evidence.
statnews.com/2024/07/29/sare…
Philipp Beck retweeted
$SRPT surprised to learn from Roche earnings that DMD gene therapy Elevidys approved in 6 ex-US countries with sales of $32M.
Philipp Beck retweeted
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
Philipp Beck retweeted
Today we report in @natBME 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.
drive.google.com/file/d/1A7w…
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