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Showing posts with label Verve. Show all posts
Showing posts with label Verve. Show all posts

Thursday, August 28, 2025

BEAM Therapeutics - A simplified investor report!

 


Beam Therapeutics (NASDAQ: BEAM)

Simplified Investor Report – August 2025


1. What Beam Does

Beam is a gene-editing company that uses a next-gen technology called base editing.
Unlike traditional CRISPR, which “cuts” DNA, base editing changes single letters of DNA directly. This makes edits potentially safer and more precise.

Beam’s approach is being tested in blood diseases, liver diseases, and cancer cell therapies.


2. Current Pipeline (Key Drugs in Development)

  • BEAM-101 (Sickle Cell Disease / Beta Thalassemia)

    • Ex vivo (patient’s stem cells edited outside body).

    • Goal: Increase fetal hemoglobin to prevent sickling.

    • FDA gave RMAT designation in Aug 2025—can speed approval.

    • Competes with CRISPR Tx’s already-approved Casgevy.

  • BEAM-302 (Alpha-1 Antitrypsin Deficiency, AATD – In vivo)

    • Liver-targeted using LNPs (same delivery as mRNA vaccines).

    • First clinical proof: restored missing protein in patients. Big de-risking milestone.

  • BEAM-301 (GSD-Ia, rare metabolic disease – In vivo)

    • First patients dosed in 2025. Very rare disease but clear genetic target.

  • BEAM-201 (Allogeneic CAR-T for T-cell leukemia)

    • First multiplex-edited CAR-T (4 edits in one cell).

    • Potential for “off-the-shelf” cancer treatment.

Why this matters: 

Beam is not a “one trick pony”—it has 4 very different programs in the clinic (blood, liver, metabolic, cancer). This spreads risk.


3. How Beam Compares to Other Gene Editors

CompanyTechnologyStatusNotes
Beam (BEAM)Base editing4 clinical programs (in-vivo + ex-vivo + CAR-T)Broadest pipeline; proof of editing in humans (AATD).
CRISPR Tx (CRSP)CRISPR/Cas9First FDA-approved therapy (Casgevy)First mover, but rollout is slow & complex.
Intellia (NTLA)CRISPR/Cas9ATTR program in Phase 3Closest to an in-vivo commercial therapy.
Editas (EDIT)CRISPRHb disorders in Phase 2Smaller player; similar to CRSP but later.
Verve (VERV)Base editingLDL cholesterol lowering in Phase 1Early but strong human data.
Prime Medicine (PRME)Prime editingFirst patients dosedVery early stage, but big potential.

Takeaway: Beam sits between CRSP’s proven approval and Intellia’s Phase 3 lead, but has more breadth than most others. Verve and Prime validate the editing class, but are earlier.

Ed Note:  We like all six and are currently invested in two of these names!


4. Why Analysts & Institutions Are Bullish

  • Analysts: 12–13 Buys, targets $40–80 (2–4x upside from ~$17 today).

  • Catalysts: RMAT for BEAM-101, clinical proof in BEAM-302, first patients dosed in BEAM-301.

  • Big investors buying: ARK Invest, Vanguard, Swiss National Bank, others.

  • Upside potential: If one of Beam’s in-vivo therapies works, it could transform rare disease treatment—making Beam a takeover target for big pharma.


5. Risks to Consider

  • Still pre-revenue—cash burn is high.

  • Competes against already-approved products (Casgevy in SCD).

  • In-vivo gene editing is very new—long-term safety still being proven.

  • Stock is volatile and heavily tied to trial readouts.


6. Bottom Line for Retail Investors

Beam is a high-risk, high-reward biotech.

  • It has a broad and diverse pipeline (blood, liver, rare metabolic disease, CAR-T).

  • Analysts see 2–4x upside in the next 12–24 months.

  • Beam is not yet proven commercially, but recent trial wins (esp. BEAM-302) show that base editing works in humans.

  • For investors who can tolerate volatility, Beam is one of the most exciting gene-editing plays—but it should be sized carefully in a portfolio.

Ed Note:  We are long BEAM NTLA CRSP EDIT



Friday, May 24, 2024

Editas Medicine and CRISPR Therapeutics are both gene-editing companies that focus on developing therapies using CRISPR-Cas9 technology.

  Let’s explore their differences and where they stand in terms of DNA editing technology:


  1. Editas Medicine:

  2. CRISPR Therapeutics:

  3. CRISPR Therapeutics is further along in terms of clinical-stage development, with data on its key gene therapy candidate already available. Given its potential market reach and financial stability, CRISPR appears to be a safer choice for investors. 
  4. However, in the long term, it’s challenging to predict which company will ultimately be the better stock. 
  5. Both companies contribute to the exciting field of DNA editing technology, and their progress is closely watched by investors and researchers alike. 
  6. Editas is now followed by a number of analysts including, Citigroup, Morgan Stanley and Barclays. These three have price targets between $9 and $15 per share at this writing!
  7. While Editas Medicine and CRISPR Therapeutics are both prominent players in the field of gene editing technology. Let’s explore some of their competitors