Philipp Beck

Drug development scientist, mRNA + AAV formulation & delivery. Works at ABL/Ascend. Enthusiastic cook & runner, daily bike commuter.

[Philipp Beck @Pilldipp]

@RNAiAnalyst Just eyeballing: deleting the top and low performer, the analysis may result in a horizontal correlation line.

[Philipp Beck @Pilldipp]

@RNAiAnalyst @zhaoweiasu Why specifically that latter fragment?

[Philipp Beck @Pilldipp]

@RNAiAnalyst That’s why it’s important to:
➡️ pick a plasmid system that generates less packaged hcDNA
➡️check your packaged impurities with NGS.

Potency (and control of impurities) should be built into the product & process from the beginning.

[Philipp Beck @Pilldipp]

@RNAiAnalyst @mike98572986 Indeed makes no sense. Much more sensible would be solid data.

[Philipp Beck @Pilldipp]

@RNAiAnalyst I found the a to I change was the most limiting bit (apart from delivery). But indeed: we perform sequence optimization all the time to optimize eg mRNA translation performance. Easy to imagine that being done for A to I

[Philipp Beck @Pilldipp]

@RNAiAnalyst SubQ I meant to write

[Philipp Beck @Pilldipp]

@RNAiAnalyst I have delivery questions. Will have to dive deeper into the background, but quarterly IT admin is challenging.
Then I read LNP delivery… which is fine, but I thought a promise of ADAR was that one might deliver barebones nucleic acid (stabilized, targeted) subs…

[Philipp Beck @Pilldipp]

@RNAiAnalyst Based the robustness of upregulation eve at 1.5 mg/kg, are they considering an even lower dose? E.g. matching the 0.3-0.6 mg/kg used by verve in heart-2?

[Philipp Beck @Pilldipp]

@medstudentinvst @adamfeuerstein In your graph at the 12 months time point is there a statistically significant difference between control and treatment arm?
What do you think about the observed trend in the control arm until month nine, that at the 12 months time point suddenly breaks?

[Philipp Beck @Pilldipp]

@RNAiAnalyst The dose that was administered to the person whose death was reported is not known yet as far as I know. In general, the Elevidys dose is at least 1.8 fold higher. All LGMD dose levels are in E13 range

[Philipp Beck @Pilldipp]

@Prof_Oak_ I would add saturdays late breaking abstract with the AAV-mediated RNA-Editing for DMD (exon skipping).

[Philipp Beck @Pilldipp]

@bradloncar Makes sense 👌

Medigene would have been my other pick, but they filed for insolvency unfortunately. Morphosys is not with us any more as well 😨

[Philipp Beck @Pilldipp]

@bradloncar Ethris because of their rich underdog-history in mRNA/LNP: since 2012 they partnered with Pharma here/there, but are now developing their own clinical product
Tubulis because they recently secured a deal with Gilead with their exciting ADC tech.

[Philipp Beck @Pilldipp]

@bradloncar Personally, I would like to hear from Tubulis & Ethris, both located in the southwest of Munich near our local biotech hub Bio-M.

Aside from that, I could imagine that our own company might be interested in welcoming you as well if you like to sprinkle your visit with AAV-lore

[Philipp Beck @Pilldipp]

@RNAiAnalyst One might see a parallel in Beam/WVE vs Hemgenix;Roctavian;Beqvez/Hympavzi I.e. non-permanent approach was preferred by the market.

[Philipp Beck @Pilldipp]

Bye twitter.

[Philipp Beck @Pilldipp]

@RNAiAnalyst I will follow your example Dirk. Where can I find you?

[Philipp Beck @Pilldipp]

@BowTiedBiotech Wow, Paxlovid! I would have not thought that this is such a value driver…

[Philipp Beck @Pilldipp]

@BuzzJacobs @bradloncar Hmmm I thought one of FDAs stances on clinical trials from china was:
A) Population in US much more diverse, thus effect of drugs must be tested in that population
B) Standard of care (comparator) in US clinical trials is harder to beat compared to chinese.