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Showing posts with label BEAM Therapeutics. Show all posts
Showing posts with label BEAM Therapeutics. Show all posts

Thursday, September 25, 2025

Today, I revisited BEAM Therapeutics and noted institutional investors increasing their positions! Here's the lowdown!

Quite a few funds besides Peregrine (17% this month) boosted their positions in BEAM Therapeutics in the last ~3 months. 

The biggest disclosed step-ups were by FMR LLC (Fidelity) and ARK Investment Management, both via fresh 13G/A filings in August 2025. I’ve also included several smaller 13F increases.




Notable increases in the past ~3 months

  • FMR LLC (Fidelity) filed a 13G/A on Aug 6, 2025, lifting its stake to 15.00% (15,083,498 sh)—a large increase from prior. Fintel

  • ARK Investment Management filed a 13G/A on Aug 7, 2025, lifting to 10.35% (10,410,137 sh). Fintel

  • Legal & General Group Plc (13F, filed Aug 12, 2025) reported 102,646 sh, up +8.48% q/q. Fintel

  • VanEck Associates (13F, filed Aug 14, 2025) reported 1,451 sh, up +11.27% (tiny but technically an increase). Fintel

  • AMG National Trust Bank (13F, filed Aug 4, 2025) reported 17,007 sh, up +289% q/q. Fintel

(And yes, Peregrine Investment Management disclosed a +17.8% increase (to 264,260 sh) in its latest 13F, covered by MarketBeat on Sept 24, 2025. ) MarketBeat

Note: 13G/A stakes (FMR, ARK) are the cleanest % of shares-out data. 13F changes indicate direction q/q but do not include exact ownership % of the company and lag by up to ~45 days.

“All” institutional investors & their % stakes

There are roughly ~455 institutional holders on record for BEAM. Publishing all of them (with % stakes) doesn’t fit here, but below are the principal holders with the best available % figures from recent filings. For >5% owners, I use the 13G/A numbers (most authoritative for company %); for others I cite aggregated holder pages that estimate % of shares outstanding.

Top institutional holders (latest reported % of shares outstanding):

  • FMR LLC (Fidelity)15.00% (13G/A, Aug 6, 2025). Fintel

  • ARK Investment Management LLC10.35% (13G/A, Aug 7, 2025). Fintel

  • Farallon Capital Management~9.99% (13G/A, May 13, 2025; still current in Fintel roster). Fintel

  • The Vanguard Group, Inc.~9.88% (aggregator snapshot). Investing.com

  • BlackRock, Inc.~8.14% (aggregator snapshot). Investing.com

  • State Street Global Advisors~3.70% (aggregator snapshot). Investing.com

  • Arch Venture Partners~4.62% (aggregator snapshot; also on Fintel 13D/G roster). Investing.com+1

  • Amova Asset Management~4.81% (aggregator snapshot). Investing.com

  • Farallon Healthcare Partners~5.87% (aggregator snapshot). Investing.com

  • Bellevue Asset Management~2.94% (aggregator snapshot). Investing.com

  • T. Rowe Price Group~2.49% (aggregator snapshot). Investing.com

For the full institutional roster (hundreds of entries) with adds/cuts and effective dates, Fintel’s BEAM ownership page is the most complete live index and shows both the headline owner count (~455) and recent filings; many details beyond the top tier are behind their login/paywall. Fintel

Ed Note:

We have also increased our position in BEAM in September!

Related Articles:

BEAM Therapeutics getting closer to FDA approvals for cutting edge therapies


Tuesday, July 15, 2025

An updated report (as of July 15 2025) on Beam Therapeutics (NASDAQ: BEAM):

 



๐Ÿ”ฌ 1. Technology Advances & Pipeline

  • Base editing leadership: Beam continues refining its precision base editing platform, enabling single-base modifications without DNA breaks—a safer alternative to traditional CRISPR/Cas9 

  • Key programs

  • BEAM‑101 (sickle cell disease): BEACON Phase 1/2 trial—26 patients dosed; all reported > 60% HbF and < 40% HbS with durable response up to 15 months; safety consistent with busulfan conditioning 

  • BEAM‑302 (AATD): IND cleared March 2025; RMAT designation granted May 2025. 60 mg cohort showed durable base-editing with therapeutic AAT levels (12.4 ยตM) and Z-AAT reduction ~78%

  • BEAM‑301 (GSD Ia): Dosing initiated Q1 2025; preclinical data support metabolic correction in animal models 

  • Platform extensions: ESCAPE non-genotoxic conditioning and pipeline expansions into CAR‑T (BEAM‑201) and eye, liver genetic targets


๐Ÿฅ 2. FDA Approval Horizon

  • BEAM‑101: Orphan Drug Designation granted June 2025; pivotal Phase 1/2 readout expected H2 2025 at EHA; BLA filing potential in 2026 if data confirm benefit and safety 

  • BEAM‑302: RMAT status enables accelerated development; next data update for dose-escalation cohort expected H2 2025; Part B (liver subgroup) also in H2 2025 

  • BEAM‑301 & ESCAPE: Phase 1 for GSD Ia and ESCAPE conditioning expected in late 2025 or 2026—these will broaden indication portfolio.


๐Ÿฆ  3. Diseases Addressed

  • Sickle Cell Disease (SCD): One-time, potentially curative therapy—addresses root cause via fetal hemoglobin induction.

  • Alpha‑1 Antitrypsin Deficiency (AATD): Base editing corrects PiZ mutation—targets both liver and lung disease where current therapies are protein replacements only.

  • Glycogen Storage Disease type Ia: Preclinical in vivo data indicate potential for metabolic correction after a single administration.

  • Cancer & Beyond: Preclinical work in CAR‑T and ocular or hepatic genetic diseases, showing platform versatility.


๐Ÿ’ฐ 4. Financials

  • Cash position: ~$1.2 billion as of Q1 2025, including $500 million public raise—runway likely into 2028 supporting key catalysts 

  • Burn rate: Q1 R&D spend ~$98.8 M, net loss ~$109 M—no revenues yet; manageable given cash reserves Stock Titan.

  • Analyst sentiment: 11 Buy, 2 Strong Buy, 2 Hold; average target ~$48.75 (range $26–$75+) MarketBeat.

  • Institutional backing: GAMMA Investing boosted awareness; over 99% public float held by institutions .


๐Ÿค 5. Partnerships & Acquisitions

  • Pfizer collaboration (since 2022): Multi-target in vivo base editing alliance.

  • Early-stage acquisition (July 1, 2025): Acquired a life sciences firm via 403,128 BEAM shares + up to $89 M in milestones—strengthens platform & pipeline BioSpace

  • Past deal: Verve/Lilly option monetization included $250 M upfront—padding runway and showing tech licensing potential .


๐Ÿ” 6. M&A Considerations

  • Takeover potential: With transformative in vivo capabilities and FDA RMAT designations, BEAM is an attractive target

  • Expect interest from Pfizer (existing partner) or large pharmas like Roche, Novartis, Regeneron.

  • Strategic fit: Platform complements existing gene therapy portfolios; acquisition could occur post-positive H2 2025 data or before pivotal Phase 3 start.


⚠️ 7. Risks & Considerations

  • Execution risk: Base editing is relatively new; long-term safety (off-target effects) remains under watch 

  • Regulatory hurdles: Despite RMAT/Odd status, full approval pathways require confirmatory data and large-scale trials.

  • Cash burn: Though well-funded now, multiple programs may drive need for additional raises (potential dilution).

  • Competition: Rivals like Vertex/CRSP in ex vivo SCD and other in vivo CRISPR players (Intellia, Editas, Verve) are active; execution differentiation is crucial.


๐Ÿ“… 8. Timeline & Key Catalysts

  • H2 2025:

    • BEAM‑101 data in adult + adolescent cohorts at EHA.

    • BEAM‑302 dose‑escalation data and Part B launch.

  • Late 2025–2026:

    • BEAM‑101 BLA prep or initiation of pivotal trials.

    • INDs for BEAM‑301 & ESCAPE platform, expanding into metabolic disease and non-genotoxic conditioning.


Update August 21 2025

Here’s the latest market snapshot for Beam Therapeutics Inc. (BEAM):

Open16.07
Volume560.7K
Day Low15.61
Day High16.40
Year Low13.53
Year High35.25

Key Developments, summer 2025

1. Institutional Moves — ARK Investment & Vanguard

  • ARK Investment (led by Cathie Wood) acquired 395,097 shares of Beam, valued at approximately $7 million on August 18, 2025, signaling continued conviction in genomic and gene-editing firms.Yahoo Finance+15AInvest+15Investing.com+15

  • Meanwhile, Vanguard Group boosted its stake in Beam by 16.4% in Q1, now holding roughly 8.87 million shares, equating to about 8.82% of the company—worth an estimated $173 million.Defense World

2. Analyst Ratings & Price Target Revisions

  • Cantor Fitzgerald reiterated an Overweight rating, with price targets ranging from $20 to $80. Their revised financial model includes updated pipelines for BEAM‑101 (sickle cell disease), BEAM‑302 (alpha‑1 antitrypsin deficiency), and BEAM‑301 (GSD‑Ia).Beam Financial+13Investing.com+13Reuters+13

  • JonesTrading, on the other hand, lowered its price target from $34 to $25, reflecting more cautious expectations.Yahoo Finance

3. Clinical & Regulatory Milestones

  • The U.S. FDA granted RMAT designation to BEAM‑101 (for sickle cell disease)—an important regulatory milestone that may streamline development and enable priority review.BioSpace+7Beam Financial+7Stock Titan+7

  • Q2 2025 financial and clinical highlights:

    • BEAM‑302 (AATD): Progress in patient dosing; strong data from earlier cohorts; well‑tolerated; proceeding into Part B in H2 2025.Beam Financial+1Investing.com+3Stock Titan+3Stock Titan+3

    • BEAM‑101 (BEACON Phase I/II for SCD): Completed dosing in 30 patients; data from 17 show promising efficacy—e.g., increased fetal hemoglobin (>60%), reduced sickle hemoglobin (<40%), durable responses, rapid engraftment, and no vaso‑occlusive crises observed post‑treatment. Additional data expected later in 2025.Stock Titan+3Stock Titan+3Investing.com+3

    • Cash position: Healthy runway with ~US$1.2 billion in cash and equivalents, supporting operations through at least 2028.Stock Titan


Summary Table — What’s New?

CategoryDetail
Institutional ActivityARK adds ~395K shares; Vanguard boosts stake by 16.4%.
Analyst SentimentMixed: Cantor Fitzgerald bullish ($20–$80 target); JonesTrading more cautious ($25 target).
RegulatoryBEAM-101 receives RMAT designation from FDA.
Clinical ProgressPositive early data from BEAM-101 and BEAM-302; enrollment nearing completion and data expected by year-end.
FinancialsStrong cash reserves (~US$1.2B), securing runway toward 2028.

What to Watch Next

  1. Further Clinical Data Releases — Additional BEACON trial outcomes for BEAM-101 expected by year-end could be pivotal.

  2. BEAM-302 Trial Expansion — Monitoring Part B initiation and data in H2 2025.

  3. Updated Analyst Reports — Future revisions from Cantor Fitzgerald, JonesTrading, and others may reflect evolving clinical timelines or financial projections.

  4. Institutional Activity — Continued moves by prominent investors like ARK and Vanguard can signal broader market confidence

Conclusion

Beam stands at a pivotal point—advanced base editing tech, promising trial data, strong financials, and regulatory momentum mean a breakout is plausible. If H2 2025 milestones are met, valuation could advance significantly, whether via commercial execution or strategic acquisition. However, precision in delivery, scalability, and long-term safety remain decisive.

Monday, June 16, 2025

How BEAM Therapeutics Base editing technology and patents are validated by the treatment of Baby KJ

 


A groundbreaking treatment that saved baby KJ earlier in 2025 used base editing—a precise form of gene editing—developed through the partnership of IGI and CHOP/Penn in collaboration with researchers at the University of Pennsylvania and Children’s Hospital of Philadelphia.

KJ was born last August with a rare genetic disorder called carbamoyl phosphate synthetase I (CPSI) deficiency, which caused him to spend the first year of his life in the hospital. KJ  is the first patient of customized gene-editing therapy.

๐Ÿ”ฌ Key Details:

  • Patient: A 1-year-old named KJ, diagnosed with Wolman disease, a fatal genetic disorder.

  • Technology Used: Base editing—a more precise and less disruptive form of CRISPR that changes a single DNA letter without cutting the DNA strand.

  • Company involved: Danaher & its IGI partnership provided the manufacturing infrastructure

  • Event timing: Treatment occurred in early 2025, reported publicly in May 2025 via The Washington Post, TIME, and Wall Street Journal.

  • How it worked: Doctors used base editing ex vivo (outside the body) on KJ’s stem cells to correct a mutation in the LIPA gene. The edited cells were then transplanted back.

๐Ÿ’ก Why It Matters:

This was the first-ever compassionate use case of base editing in a human patient and potentially the first customized CRISPR-derived therapy to save a life in real-time clinical crisis—proving that IGI's and  Beam's technology as not just experimental, but life-saving.


What it means for our investment in BEAM Therapeutics:

Pipeline Acceleration & Clinical Validation

1. BEAM‑302 (AATD) – Confirmed Efficacy & Safety

  • Early Phase 1/2 results show dose-dependent increases in functional AAT protein and up to 78% reduction of mutant protein after a single dose Analysts see this as a “bar-setter” for in vivo base editing, especially since LNPs (used for liver delivery) showed a clean safety profile, easing concerns from related therapies 

2. BEAM‑101 (Sickle Cell Disease)

  • The ex vivo base editing trial has now treated 17 patients, with updated safety and HbF efficacy data presented at EHA 2025 

  • Beam plans to complete dosing in 30 patients by mid-2025 

3. BEAM‑301 (GSD Ia)

  • A Phase 1/2 trial began in May 2025, marking the expansion into metabolic/liver-focused in vivo editing 

4. ESCAPE Conditioning Platform

  • Progress continues on a non-genotoxic conditioning strategy to improve stem cell transplants, supporting broader deployment of ex vivo therapies

๐Ÿ’ฐ Investment & Financial Health

  • In March 2025, Beam raised $500M through a follow-on offering at $28.48/share to specifically fund BEAM‑101, BEAM‑302, and ESCAPE 

  • As of Q1 2025, Beam holds approximately $1.2 billion in cash, sufficient to support operations into 2028 


๐Ÿ“ˆ Analyst Sentiment & Stock Outlook

  • Analyst consensus is generally bullish, with ratings like “Buy/Outperform” and median price targets near $46 (50–100% upside from current levels) Key near-term stock catalysts include:id-2025 data readouts from BEAM‑101 and BEAM‑302.

    • Late‑2026 goal for BEAM‑101 Biologics License Application (BLA) filing 

⚖️ Risk vs. Reward

UpsideRisks
• One-time, durable cures• High R&D spending; cash burn continues 
• Competitive, less invasive delivery• Potential off-target effects or regulatory delays
• Strong capital & novel platform• Market adoption and reimbursement uncertainties

✅ Summary:

Beam’s pipeline is more validated and better funded than ever:

  • In vivo successes in AATD (BEAM‑302) and GSD Ia (BEAM‑301).

  • Ex vivo progress in BEAM‑101 (SCD).

  • Extensive cash runway, likely enough to reach major clinical catalysts.

  • Investor optimism keyed to upcoming data and potential BLA filings.

๐Ÿ‘‰ Bottom line: Beam is positioned as a high-risk, high-reward play in genetic medicine—investor visibility has increased dramatically in 2025 with clinical proof-of-concept and strong funding. 

The next 6‑12 months of data releases will be crucial.

ED Note:  We are long BEAM stock, and, we would not be surprised if their was a buyout!

Let's review the details once again!

1. IGI and CHOP/Penn developed the CRISPR base-editing therapy

  • The Innovative Genomics Institute (IGI) at UC Berkeley, led by Jennifer Doudna, Fyodor Urnov, and Petros Giannikopoulos, performed the key research: identifying the base-editing approach, designing the editor and guide RNA, running safety assays, and helping secure FDA approval. They collaborated closely with CHOP and Penn clinical teams to deploy the therapy to Baby KJ statnews.com+7vcresearch.berkeley.edu+7the-scientist.com+7.


๐Ÿš€ 2. Danaher & its IGI partnership provided the manufacturing infrastructure

  • IGI had formed the “Danaher‑IGI Beacon for CRISPR Cures” in early 2024—a collaboration between IGI and Danaher to build an on‑demand “cookbook” and scalable manufacturing pipeline for CRISPR therapies danaher.com+6innovativegenomics.org+6genengnews.com+6.

  • When KJ’s case emerged, Danaher’s DBS (Danaher Business System) activated its subsidiaries—IDT (guide RNA), Aldevron (mRNA editor), Acuitas (LNP delivery)—to rapidly produce the therapy in under six months (instead of 18–24), a critical logistical feat genengnews.com+5danaher.com+5the-scientist.com+5.


๐Ÿ”„ How these threads intersect

RoleEntityContribution
Research & designIGI + CHOP/PennIdentified mutation, designed editor, performed safety testing, architected therapy
Manufacturing & scale-upDanaher via Beacon/DBSRapid production of therapeutic components and delivery vehicles through its subsidiaries

๐Ÿ‘ถ So what really happened with Baby KJ?

  • The scientific breakthrough—the precision base-editor treatment—came from IGI, CHOP, and Penn.

  • The “turning it into a real drug, fast enough to treat KJ” depended on Danaher’s industrial firepower through the Beacon collaboration and DBS efficiencies.


Both aspects are essential: the molecular innovation (from IGI/academia) and the manufacturing/logistical execution (from Danaher).
That’s why some reports emphasize the science from BEAM Therapeutics (or rather, IGI-led research), while others highlight the Danaher-IGI Beacon and DBS manufacturing.


✅ Bottom line

  • IGI/BEAM side: provided the underlying gene-editing tech and clinical strategy.

  • Beacon/ Danaher side: supplied the infrastructure to make, test, and deliver it rapidly.

They worked hand-in-hand—that’s why both narratives appear, and why both are true.

The base-editing technology used to treat Baby KJ originated from academic research led by the Innovative Genomics Institute (IGI)—not BEAM Therapeutics—though Beam works on similar tech.

Here’s a breakdown of who owns and developed what:


๐Ÿงฌ The Core Technology:

  • Developed by IGI scientists, especially at UC Berkeley, led by Dr. Fyodor Urnov and Dr. Jennifer Doudna.

  • The therapy used adenine base editing (ABE) to correct a single-letter mutation in the SLC17A5 gene.

  • The editor, guide RNA, and delivery method were designed and validated in-house at IGI—not licensed from Beam.


๐Ÿ”ง Who Actually Made It:

  • IGI didn't have manufacturing capacity, so they partnered with Danaher through the Danaher-IGI Beacon for CRISPR Cures.

  • Danaher subsidiaries—like Aldevron (mRNA), IDT (guide RNA), and Acuitas (LNP)—produced the treatment materials.

  • Danaher enabled IGI’s therapy to reach the clinic fast, but did not invent the core editing tech.


๐Ÿงช What About Beam Therapeutics?

  • "Beam popularized and refined base editing commercially and owns key patents".

  • However, Beam was not involved in Baby KJ’s case.

  • That said, Beam uses a similar base-editing approach and often collaborates with academic groups, but this specific case was independent of Beam.


✅ Final Answer:

The technology used to cure Baby KJ was developed by IGI scientists at UC Berkeley, not Beam Therapeutics.
Danaher provided manufacturing and scale-up support via its Beacon collaboration—but IGI owns the scientific credit.

Here's a structured analysis of how the successful treatment of Baby KJ using IGI-developed base editing impacts Beam Therapeutics' (BEAM) pipeline and investment outlook:


๐Ÿ“ˆ 1. Validation of Base Editing as Clinically Viable

While Beam Therapeutics wasn’t involved in Baby KJ’s treatment, the successful real-world application of base editing dramatically validates Beam’s entire platform:

SignalImplication
Adenine base editing corrected a lethal mutation in a real patientConfirms the precision, safety, and efficacy of base editing
FDA allowed compassionate useBoosts credibility with regulators, paving the way for Beam’s future INDs
Global media & scientific attentionRaises investor awareness and optimism for Beam’s pipeline success

๐Ÿ” Bottom line: This proves that base editors can go beyond the lab—a major credibility boost for Beam.


๐Ÿ”ฌ 2. Beam’s Pipeline Benefits from Timing and Similarity

Beam’s current lead programs are in genetic blood and liver disorders, which also involve single-nucleotide mutations—perfect use cases for base editing:

ProgramTargetStage
BEAM-101Sickle cell diseasePhase 1/2
BEAM-302Alpha-1 antitrypsin deficiency (AATD)IND submitted
BEAM-301Glycogen storage disease IaIND-enabling
  • Many of these programs use adenine base editors, just like in KJ’s case.

  • The delivery mechanism (LNPs) and editor format (mRNA + guide RNA) are the same—so clinical translation is de-risked.

๐Ÿ’ก Translation advantage: Beam now benefits from clinical proof-of-concept, without taking the risk themselves.


๐Ÿ’ฐ 3. Investor Confidence & Potential Catalysts

FactorImpact
Base editing now proven feasibleIncreases likelihood of Beam’s trials succeeding
Beam owns extensive IP on base editorsCould lead to licensing opportunities or acquisitions
Current price (~multi-year lows)Might attract deep-value and biotech-focused funds
Potential M&A targetLarge pharmas (e.g., Pfizer, Roche, Vertex) may now see Beam as a validated platform rather than early-stage speculation

๐Ÿง  Expect analyst upgrades, stronger buy-side attention, and possibly strategic interest in Beam due to this milestone.


๐Ÿ“‰ Risk Factors Still Exist

RiskNote
Beam didn’t do the KJ trialNo direct clinical validation of their own programs yet
Competition from IGI, Verve, EditasAcademic groups or biotechs could move faster in specific niches
Delivery & durability remain challengesEspecially for systemic delivery beyond liver or bone marrow

๐Ÿง  Investment Takeaway

Even though Beam didn’t treat Baby KJ, the entire biotech world now knows base editing works in humans. That reduces risk for Beam’s clinical pipeline, increases their perceived value, and makes them a more attractive investment or acquisition target.

This event has quietly become one of the most important validations of Beam’s thesis—and it happened without them needing to spend years in the clinic.

ED Notes:

Full disclosure: I am long both BEAM Therapeutics and Editas as well as 

CRSPR and NVDA (not mentioned here)