zeus

Wish everyone a happy holiday and prosperous 2025. My speculative bets on small biotech for 2025 are $SGMO and $INMB. My long-term holds are $NTLA and $MRNA. My sure bet is on TIPS (Treasury Inflation-Protected Securities).

@XueJia24682 Total lies.

✨🇨🇳In China, senior citizens enjoy government support. Each meal costs only about $0.70, subsidized by the state. They also receive an additional monthly living allowance, and door-to-door meal delivery is available.

$SGMO owns valuable technologies. Will it survive the current money crunch and realize its potential? It’s has been painful to own the stock.

Incredibly relevant to the times we live in

@BiotechAnalysst $SGMO The initial plan is to use ZFA (Zinc Finger Activator) to activate the good copy of the gene (on X chromosome). STAC-BBB delivers ZFA to all neurons.

#Astellas not buying $TSHA re but $SGMO ?

@BiotechAnalysst Exactly! Delivery to the brain without general anesthesia and 10+ hours of surgery. It is possible to halt the disease completely!

what if there was a possibility to #delivery therapies into all regions of the brain #without #surgery ? wouldnt that be worth billions ? re $SGMO #STAC_BBB

During a gathering with friends past weekend, one asked the question of what was it like before the Big Bang? I answered him that there was no “before” the Big Bang. Time and space only started with the Big Bang!

Efficacy, ease of administration, and once-and-done will make $NTLA products the standards of care for HAE and ATTR. Annual sales will be more $10B by 2034. What should be the current valuation?

Overexpress proteins in CNS will always be challenging. Immune response in CNS is difficult to manage. Suppression of proteins should not have similar issues (Tau, SYN, PRP). Capsida, $VYGR, $SGMO. Look forward to clinical testing on PRP suppression.

$SGMO Early treatment with ST-920 is key to restore kidney function. Patients with mild kidney damage (eGFR>60) show the most improvement. This is further supported by results from female patients. Miracle drug for Fabry patients!

@Franca_ole $SGMO ST-920 exemplifies the critical importance of a safe AAV vector. ST-920 overexpresses alpha-GalA up to over 100x physiological concentrations, which enable the reversal of disease to certain extent. Strict correction of the mutation in the genome will not be able to.

Early detection is important – gene therapy with $SGMO ST-920 cures! Fabry disease is an X-linked lysosomal storage disorder in which α-galactosidase A deficiency leads to the accumulation of globotriaosylceramide (Gb3) in many tissues, including heart tissue. This leads to hypertrophy (thickening of the myocardium), fibrosis (scarring), arrhythmias and heart failure. Cardiac involvement can begin early, often before it is visible on an ECG or imaging. hs-cTn is a highly sensitive cardiac troponin or protein from heart muscle cells. Even very slight cell damage leads to a measurable increase in troponin in the blood. In genetically confirmed FD, serial hs-cTn measurement allows for the very early detection of myocardial damage, often years before structural changes become visible. This allows timely therapeutic intervention to prevent or slow down heart damage. onlinelibrary.wiley.com/doi/10.1002/jm…

$SGMO Fabry deal will be done within 4 weeks. The upfront money will be less than expected (SGMO demands for 2 years of funding of ~250 to 300M). Now upfront will be ~150M with approval of additional $80M. ~15% royalty.

@BiotechAnalysst @yusufhameed @ZinselmeyerB AAV vector type, production quality, targeting protein and its required expression level. Among them, dose (amount of AAV) is the most critical. Reasonable dose is pretty safe for most AAV vectors including AAV2/6. AAV5, AAV7, and AAV9.

@BiotechAnalysst @yusufhameed @ZinselmeyerB It is always a challenge to target muscle cells. The newer AAV vectors from $SGMO, especially tranferin receptor binding one, could be advantageous. On the other hand, there are many factors impacting immune response.

$SRPT should contact $SGMO ASAP for a better delivery option #STAC

@davidrliu @CellCellPress Better brain targeting vectors such as STAC-BBB could make it more efficient and less side-effect, especially liver toxicity. Impressive! $SGMO $PRME

Today in @CellCellPress we report the use of prime editing to correct several mutations that cause alternating hemiplegia of childhood (AHC), a rare and devastating neurodevelopmental disorder, in patient-derived cells and in two mouse models. drive.google.com/file/d/1ibC50t… 1/10

@drsprs @adamfeuerstein $NGNE 3x10^15 vg AAV9 is just too much. Why not use newer generations of AAV9 from $VYGR or $SGMO. Might be able to use 100x less vg to achieve comparable neuronal expression.

@adamfeuerstein Interesting that girl got 3x10^15 and registrational protocol dose is 1x10^15. Still high-ish.

You can kill a patient and still get an easy pass from FDA on a Phase 3 study, apparently. $NGNE No lesson learned from $SRPT?

@RepMarkPocan It's Fat and Ugly Bill.

My flight to DC was canceled so I'm driving to Chicago to catch a 6AM flight because I refuse to miss voting NO on the Big Ugly Bill!

@BradleySteveson In your opinion, positive results?

@zeusPharma Easy. They probably want/need the weekend to polish up their material for the call.

$INMB Can anyone analyze the psychology for Monday morning? If totally negative, Friday after hours would be perfect. If blown away positive, why wait for 3 days?

$SGMO As the median age of patients is in their 40s, most of them will gain back about ~5 years of kidney function in terms of eGFR after two years. The general population loses about 6 to 7 points between the age 40 and 50. It’s a miracle drug for Fabry patients.

$SGMO “Furthermore, a mean annualized eGFR slope of 1.747 mL/min/1.73m2/year (95% CI: -0.106, 3.601) was observed for the 19 patients who have achieved 104-weeks of follow-up” Patients gained years of kidney function after two years on treatment.

With mature data, approval is near certainty both in US and EU. $SGMO needs to get an experienced partner. It will reach peak sales of over 1B for over a decade.