1/Arbor Biotechnologies has recently announced the acquisition of Serendipity Biosciences - a private BioTech company focused on the discovery of unique gene editing technologies which were discovered in the lab of the leading CRISPR researcher & Arbor co-founder - Feng Zhang. $XBI

1/As promised and after reading $CRBU latest Q1 2024 financial report here is my impression regarding Caribou Biosciences latest corporate status. As always - I have focused only on the main issues that I found to be the most interesting & relevant. Here’s my summary 🧵👇 $XBI #BioTech

Casgevy-grade-impressive data from Intellia Therapeutics on durability and clinical efficacy of one-time gene editing to treat HAE. CRISPR gone in 72 hrs, curative benefit persists out to 88 weeks. Congruent to their earlier data on TTR. = CRISPR is a platform for one-and-done cures.

1/Beam Therapeutics reported data highlighting its manufacturing process for its base-edited CD34+ hematopoietic stem & progenitor cell (HSPC) gene editing medicines & $BEAM-101 as part of the BEACON Phase 1/2 clinical trial in patients with severe sickle cell disease (SCD). #EHA2024 $XBI

1/WOW! A huge collaboration was announced today between Ascidian Therapeutics & Roche for the discovery & development of RNA editing-based therapeutics targeting neurological diseases. Ascidian will receive $42M in upfront payment & up to $1.8B in R&D & commercial milestones. $RHHBY $XBI

ATTR-CM (Cardiomyopathy) A Master Thread 🧵 1️⃣ Stabilizers: $PFE- Tafamidis owns current market $BBIO- appears stronger than Pfizer 2️⃣ Knockdown/TTR inhibitor: $ALNY- Helios B (data in ~2 wks) $IONS- Eplontersen (2025 readout) $NTLA- 2001 (data in ~3/4 months) 3️⃣ Removal

1/WOW! Intellia Therapeutics presented new & promising data from its Phase 1 study of $NTLA-2001 - an in vivo CRISPR/Cas9 single-dose Gene Editing therapy for treating transthyretin (ATTR) amyloidosis, which demonstrated the potential for redosing with its LNP-based delivery platform. $XBI

After looking into it for a while, I believe $BBIO offers some of the most attractive upside I've seen in a commercial stage biotech recently. A pretty long thread on why I think BridgeBio is a great investment 🧵1/

The biggest problem of Gene Editing & Gene therapy is delivery - how to deliver a genetic payload to the exact desired location. Here’s a great Infograph which shows which BioTech companies use Viral delivery methods (AAVs, LVs) compared to non-viral platforms (NLPs). $XBI

Sarepta $SRPT demanded Duchenne patient advocacy group censor video critical of the company. New story, from me. statnews.com/2024/07/29/sar…

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

1/One of the best scientific papers that I’ve read about genetic medicine is “Precision Gene editing in the eye” by Susie Suh & Elliot Choi, MD PhD in Kris Palczewski’s lab at UC Irvine & David R. Liu. It shows the huge progress made in CRISPR, Gene therapy & IRDs. Here’s my 🧵👇 $XBI

I remember how amped I was while doing this DD - same feeling for $BBIO now at these prices. Absolutely regret selling $VKTX early however

$BBIO - Giving a Master Class to biotech PR and management teams on how to press release FDA approval and conduct subsequent conference call. 1.Thank you to the clinical trial participants upfront as the first priority, backing that up with free drugs for life for those brave

Moved all of my retirement account into $IBIT at $53. Will revisit this in a year.

Stacking as many 8/15 $29 $BEAM calls as I can in preparation for the AATD readout

$KYMR + / - ~20% tomorrow?

actually think $OSCR is a good play despite the craze rn, went super long at ~$20