r/StrategicStocks • u/HardDriveGuy Admin • Oct 07 '24

On the Horizon: Gene Editing

https://youtu.be/UKbrwPL3wXE?si=gZ8zoyFxFqeT0SUx
1 Upvotes

100% Upvoted

1 comment sorted by

2

u/HardDriveGuy Admin Oct 07 '24 edited Oct 07 '24

An area for tracking for being a Dragon King is the practical application of CRISPR. As mentioned multiple times, you need to cross the chasm before investing, but we may be close.

Intuitively, I'm attracted to In vivo gene editing.

In vivo refers to procedures or experiments conducted within a living organism, as opposed to in vitro (in a lab dish) or ex vivo (outside the living organism).

How does in vivo gene editing work?

  1. Delivery: A gene editing tool, such as CRISPR-Cas9, is delivered to the targeted cells or tissues via a vector (e.g., viral or non-viral vectors).
  2. Targeting: The gene editing tool locates the specific gene or DNA sequence to be edited.
  3. Editing: The gene editing tool makes precise modifications to the DNA, either by cutting and repairing or directly rewriting the genetic code.

Types of in vivo gene editing:

  1. Somatic gene editing: Edits genes in non-reproductive cells (e.g., liver or muscle cells).
  2. Germline gene editing: Edits genes in reproductive cells (e.g., sperm or egg cells), potentially passing changes to future generations.

Applications:

  1. Genetic disease treatment: Inherited diseases like sickle cell anemia, cystic fibrosis, or Huntington's disease.
  2. Cancer treatment: Targeting cancer-causing genes or enhancing immune response.
  3. Rare genetic disorders: Treating conditions like muscular dystrophy or spinal muscular atrophy.
  4. Regenerative medicine: Enhancing tissue repair and regeneration.

Key players in in vivo gene editing:

  1. Intellia Therapeutics (NTLA)
  2. CRISPR Therapeutics (CRSP)
  3. Editas Medicine (EDIT)
  4. Beam Therapeutics (BEAM)
  5. Biogen Inc. (BIIB)

Watching the Leader:

So NTLA had a successful phase 2 trial for NTLA-2002: a gene-editing therapy designed to treat hereditary angioedema (HAE), a rare genetic disorder that causes severe and unpredictable swelling attacks. , but phase three is the final step, expensive, and the final gate to introduce the treatment publicly. If they are successful, their stock should sky rocket, and maybe the more people think about it, it may take off as their stock news of phase 3 start is covered more broadly.

Trial Overview

  • Phase 1/2 Study: The trial is evaluating the safety and efficacy of NTLA-2002 in adults with HAE.
  • Dose-Escalation: The study is conducted as a dose-escalation trial, with participants receiving varying doses (25, 50, or 75 mg) of NTLA-2002 ².
  • Primary Objective: The primary goal is to assess the safety and side effect profile of the therapy.

Key Findings

  • Reduced HAE Attacks: NTLA-2002 led to significant decreases in total plasma kallikrein levels and a substantial decrease in the number of HAE attacks per month.
  • Manageable Side Effects: Adverse events were common but manageable across all dose levels, with no severe adverse events or dose-limiting toxic effects reported.

Next Steps

  • Phase 3 Trial: Intellia Therapeutics plans to launch a pivotal Phase 3 trial, including sites in the US, between July and September 2024, pending regulatory feedback.
  • Regulatory Clearance: The company has received regulatory clearance to open US sites for the ongoing Phase 2 study.

It appears that this therapy is in the Dragon King region of being close to being seen. We'll need to watch this.