In the last few days there have been news about Sangamo Therapeutics ($SGMO (-2,28 %)), which for me are more than just headlines:

➡️ a new patent for the further development of zinc finger technology

➡️ and a sign of life from the paused TX200 program

Both show that Sangamo continues to expand its platform - quietly but steadily. ☝️

1️⃣ New patent: "Engineered Genetic Modulators"

On October 9, 2025 a new patent was published at the US Patent and Trademark Office (USPTO):

US20250313848 - "Engineered Genetic Modulators" which you can view here 👉https://tinyurl.com/3e8d2bpr

(the original patent US20200109406 from April 9, 2020 can be found here 👉 https://tinyurl.com/mry9rhxp - If the two links do not work, simply go to https://ppubs.uspto.gov/pubwebapp/static/pages/ppubsbasic.html and enter the respective patent number without the suffix "US" in the search field)

This new patent extends Sangamo's Zinc Finger Platform (ZFP) - the molecular tools the company uses to controls genesinstead of cutting them cutting them up.

While many competitors still rely on CRISPR a technology that permanently alters DNA - Sangamo has long been working with a more precise, controllable systemthat modifies genes like a dimmer instead of irrevocably rewriting them. This is an enormous safety advantage, especially in the nervous system.

In short: CRISPR cuts - Sangamo directs. And this is exactly what the next generation of patents is being created for - a new era of controllable gene therapy.

2️⃣ TX200 - the "paused" program lives on

Almost at the same time there was a sign of life on LinkedIn:

Sangamo shows there that the CAR-Treg program TX200 - developed to prevent rejection reactions after kidney transplants - is still scientifically active.

💡 The special feature: TX200 was the world's first clinically approved CAR-Treg program. Sangamo was therefore a pioneer - and made clinical history long before other players emerged in this field.

Pausing this program was not a setback, but a strategic decision: The program is expensive, complex and needs partner capital - but scientifically it is fully intact. Instead of suppressing the immune system, it is "retrained" to accept the donor organ - a completely new immunological approach.

The Nobel Prize and the fact that Sangamo is now taking up the topic again publicly shows that the platform is alive - and remains relevant: The platform is alive - and remains relevant.

🧠 Background: The zinc finger platform - Sangamo's silent backbone

If you want to understand Sangamo, you have to understand the technology. The company works with zinc finger proteins (ZFPs) - tiny DNA binders that precisely switch genes switch genes on or off, correct or regulate them. or regulate genes. Ingenious!

This results in three pillars of application:

1️⃣ Gene regulation - Switching genes on or off in fine doses (e.g. ST-503, ST-506, tauopathies with Genentech)

2️⃣ Gene editing - Correct DNA sequences permanently (e.g. sickle cell)

3️⃣ CAR-Tregs - Programming immune cells (e.g. TX200)

And in order for these therapies to reach the brain at allSangamo is also developing its STAC-BBB delivery platform - a kind of molecular transporter that carries genetic therapies across the blood-brain barrier.

This platform is unique in the industry and is already being used by:

• Genentech / Roche (tauopathies)
• Astellas (exclusive license for up to five CNS targets)
• Eli Lilly (parallel license package with up to USD 1.4 billion milestone potential)

Sangamo earns from license fees, milestones and royaltieseven when others are in the spotlight.

🎯 Conclusion - Two small pieces of news, but one big pattern:

Sangamo remains true to its line - focused, data-driven and long-term. Not a hype story, but an example, how quiet pioneers are building the future step by step.

💭 And one final thought

Now there are visible triggers - and that changes everything:

🔹 The new patent confirms the further development of the platform

🔹 TX200 shows activity again

🔹 ST-503 starts clinically

🔹 Fabry is about to be approved

🔹 Further partnerships are on the horizon

Sangamo has never had this combination before. It looks to me like the company is on the verge of a real revaluation - scientifically, strategically and perhaps now also on the stock market again 🤑😜

What fascinates me again and again: Sangamo has the technology that others are still looking for. While companies like CRISPR Therapeutics ($CRSP) (+2,19 %) or Intellia ($NTLA) (+2,08 %) are being criticized for their gene scissors ✂️gefeiert, Sangamo has long been working on the next generation - more more precise, safer, reversible. Not a one-way street in the genome, but real control.

Looking back, I think it's fair to say that Sangamo was ahead of its time - a pioneer when the world wasn't even ready. Today, everything fits together: Data, platform, partners, momentum. When the market finally recognizes this, we won't be talking about percentages - but about breakthroughs that make history. 🚀

Unfortunately I got in late, but as is the case with watchlist candidates, they have to prove themselves first.

$NANO (-4,16 %) ,

$NTLA (+2,08 %) also there!

Hello everyone,

Recently the price of $NTLA (+2,08 %) has risen significantly. Here are the most important reasons and current facts:

• Positive study data: Very strong 2-year data on the CRISPR-based gene therapy nexiguran ziclumeran (nex-z, NTLA-2001) for transthyretin amyloidosis (ATTR) was published on May 18, 2025. The treatment shows a stable and significant reduction in TTR protein by approximately 90% over 24 months and clinical improvements in patients, even in those who previously failed to respond to other therapies ([SEC source, p. 2-3]2).
• Phase 3 trials are ongoing: The pivotal Phase 3 trials (MAGNITUDE and MAGNITUDE-2) for various forms of ATTR are proceeding as planned. Recruitment for the ATTRv-PN trial (MAGNITUDE-2) is progressing well, completion of recruitment for the large ATTR-CM trial is planned for early 2027. A marketing authorization application (BLA) for nex-z is expected in 2028 ([SEC source, p. 1]2).
• Strategic focus: Intellia has focused on the most important programs in 2025 (nex-z and NTLA-2002), other projects have been put on hold. This is intended to reduce costs and increase the chances of success of the core products ([SEC source, p. 7-8]1).
• Finances: Despite high losses (Q1/2025: over USD 114m), the liquidity base is solid and, according to management, extends into the first half of 2027 ([SEC source, p. 10]1).

Links to the sources:

• SEC-10-Q Quartalsbericht vom 8. Mai 2025 (Details zu Pipeline, Finanzen, Strategie)
  1
• SEC-8-K Bericht vom 19. Mai 2025 (Studiendaten, Ausblick, Studienfortschritt)

What do you think - will the share continue to rise or is the risk too high? What is your assessment, buy now or wait and see? This is really just speculation.

https://www.sec.gov/Archives/edgar/data/1652130/000095017025066914/ntla-20250331.htm

$NTLA (+2,08 %) Does anyone have any idea how this sharp fall in the share price came about?

The company seemed to me to be very highly valued at one point, but there doesn't seem to be much left of it now.

Currently no longer a player in the pharma or CRISPR sector? 🧐

Construction of a biotech ETF🧬🚀

I'm a fan of the whole topic and don't really like the sector ETFs on the market in terms of costs and performance. That's why I've dared to construct my own "biotech ETF". I've written down my thought process here and hope you enjoy it a little 😉

1. focus areas according to growth and profitability

The percentage distribution of focus areas is based on an analysis of the growth and profitability potential of each therapeutic area within the biotechnology industry, taking into account various market conditions and dynamics. The reasons for this distribution are as follows:

• Oncology (25%): The oncology market is a large and rapidly growing market due to the high prevalence of cancer and increasing demand for innovative cancer therapies. The growth potential is supported by the continuous introduction of new cancer drugs and therapies.

• Neurology (20%): Although the neurology market is smaller compared to the oncology market, it still offers considerable potential for growth and profitability. The increasing prevalence of neurodegenerative diseases and the need for novel treatment approaches contribute to this potential.

• Immunology (15%): Immunology is an established market with a wide range of therapies for various autoimmune diseases and cancers. Although the market is relatively saturated, advances in immunotherapy continue to offer opportunities for growth and profitability.

• Infectious diseases (20%): The infectious diseases market has gained significant importance due to global health crises such as the COVID-19 pandemic. The increasing demand for vaccines, antiviral therapies and diagnostic tests to combat infectious diseases is contributing to robust growth and profitability potential.

• Cardiovascular diseases (10%): Although the cardiovascular disease market is not growing as fast as other areas, it remains a significant market with some growth potential. Advances in the treatment and prevention of cardiovascular diseases could contribute to further growth.

Genetic diseases (10%): The market for genetic diseases is smaller but highly specialized and growing thanks to advances in genomic research and genome editing technologies. The increasing demand for personalized therapies for rare genetic diseases is contributing to stable growth.

By taking into account market size, growth potential and profitability, we have determined a percentage distribution of focus areas that reflects the attractiveness of each area for investment in the Biotech ETF.

2. selection of companies and their focus areas

Based on the evaluation of the focus areas, we have selected ten biotech companies that cover a broad range of therapeutic areas and hold leading positions in their respective focus areas. The companies and their focus areas as well as the percentage distribution are as follows:

• Amgen ($AMGN (+0,53 %)):

Introduction: Amgen is a leading global biopharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies. The company is known for its breakthrough developments in oncology, immunology and genetics.

Focus areas:

Oncology: 30%

Immunology: 20%

Cardiovascular diseases: 20%

• Genetic diseases: 30%

• Regeneron Pharmaceuticals ($REGN (-0,34 %)):

Introduction: Regeneron Pharmaceuticals is a biopharmaceutical company focused on the development of drugs for serious diseases. The company is known for its pioneering research and development in the fields of oncology, neurology, immunology and infectious diseases.

Focus areas:

Oncology: 30%

Neurology: 30%

Immunology: 20%

• Infectious diseases: 20%

• Vertex Pharmaceuticals ($VRTX (-0,51 %)):

Introduction: Vertex Pharmaceuticals is a leader in the development of therapies for rare genetic diseases. The company is dedicated to researching and developing drugs to improve the lives of patients with genetic disorders.

Focus areas:

• Genetic diseases: 100%

• Intellia Therapeutics ($NTLA (+2,08 %)):

Introduction: Intellia Therapeutics is a pioneer in the field of genome editing, utilizing CRISPR/Cas9 technology to develop therapies for genetic diseases, oncology and infectious diseases. The company strives to create the next generation of gene therapies.

Focus areas:

Genetic diseases: 50%

Oncology: 30%

• Infectious diseases: 20%

• Biogen Inc. ($BIIB (+0,83 %)):

Introduction: Biogen is a leading global biotechnology company focused on the research and development of therapies for neurological diseases. The company has made some of the most significant breakthroughs in the treatment of diseases such as multiple sclerosis and Alzheimer's disease.

Focus areas:

• Neurology: 100%

• Moderna, Inc. ($MRNA (-0,55 %)):

Introduction: Moderna is an innovative biopharmaceutical company focused on the development of mRNA therapies and vaccines. The company has attracted worldwide attention for the development of an mRNA vaccine against COVID-19.

Focus areas:

Oncology: 70%

• Infectious diseases: 30%

• Illumina, Inc. ($ILMN (+0,65 %)):

Introduction: Illumina is a leading provider of genomics and genomic analysis technologies. The company is revolutionizing genomics by developing cutting-edge sequencing technologies and making significant contributions to genetic disease research.

Focus areas:

• Genetic diseases: 100%

• CRISPR Therapeutics AG ($CRSP (+2,19 %)):

Introduction: CRISPR Therapeutics is a leader in the application of CRISPR/Cas9 genome editing technologies to treat genetic diseases. The company has the potential to revolutionize the way we treat genetic diseases.

Focus areas:

• Genetic diseases: 100%

• Alexion Pharmaceuticals, Inc.$n/a): (ISIN: US0153511094)

Introduction: Alexion Pharmaceuticals is a biopharmaceutical company focused on developing therapies for rare and life-threatening diseases. The company has a strong presence in the fields of neurology and immunology.

Focus areas:

Neurology: 40%

• Immunology: 60%

• Qiagen N.V. ($QGEN):

Introduction: Qiagen is a global leader in molecular diagnostics and research products. The company is known for its innovative technologies to diagnose infectious diseases and support life science research.

Focus areas:

Infectious diseases: 100%

This selection of companies represents a diverse range of focus areas and positions in the biotechnology industry and enables

3. weighting of the ETF based on focus areas and companies

Finally, we determined the allocations for each stock based on its exposure to the focus areas and its relative importance within the portfolio. We considered both the growth and profitability potential of each focus area. The final allocation of stocks is as follows:

Regeneron Pharmaceuticals (ticker: REGN): 25%

Moderna, Inc. (Ticker: MRNA): 18%

Intellia Therapeutics (Ticker: NTLA): 16%

Amgen (Ticker: AMGN): 16%

Alexion Pharmaceuticals, Inc (Ticker: ALXN): 10%

Biogen Inc (Ticker: BIIB): 6%

Qiagen N.V. (Ticker: QGEN): 5%

Vertex Pharmaceuticals (Ticker: VRTX): 2%

Illumina, Inc. (Ticker: ILMN): 2%

CRISPR Therapeutics AG (Ticker: CRSP): 2%

This weighting ensures that the portfolio has a good balance between different focus areas as well as an appropriate exposure to the leading companies in the biotechnology industry.

By considering market size, growth potential and profitability, we have determined a percentage distribution of focus areas that reflects the attractiveness of each area for investment in the biotech ETF.

I look forward to receiving feedback and assessments ✌️😉

Your Value Willi

What is $NTLA (+2,08 %) for a company and what is my investment case?

Intellia is the stock ticker for Intellia Therapeutics, a company focused on the application of CRISPR-Cas9 technology in medicine. [1]

What is CRISPR-Cas9 technology?

CRISPR-Cas9 technology is a revolutionary biotechnology tool that enables researchers to precisely alter the genetic makeup of living things, including humans.

CRISPR: This stands for "Clustered Regularly Interspaced Short Palindromic Repeats" and refers to repeated DNA sequences found in bacteria that function like a molecular memory. These sequences help bacteria defend themselves against attacks from viruses.

Cas9: This is a protein produced by bacteria that functions like molecular scissors. It can be inserted into DNA and specifically cut or modify certain genes.

Simply put, it is a tool for precisely modifying DNA, which is also very inexpensive.

[2]

Here is a video for a clear explanation.

https://www.youtube.com/watch?v=ZAz1GutJGbg

Now for the stock:

Intellia Therapeutics has the potential to revolutionize the way genetic diseases are treated. CRISPR-Cas9 allows scientists to target genes in the human genome to cure or prevent disease. This has far-reaching implications for medicine and the healthcare industry, as it offers the possibility of treating previously incurable diseases.

Intellia is a leader in gene editing with Crispr Therapeutics.

Intellia is working with Regeneron Pharmaceuticals and already has a few projects in the "EARLY-STAGE CLINICAL Phase".

What is an EARLY-STAGE CLINICAL phase and what is special about it?

The "EARLY-STAGE CLINICAL phase" refers to the beginning of a clinical trial in which a drug or therapy is tested in human subjects. This phase is usually divided into three main stages: Phase I, Phase II, and Phase III. Each of these phases has its own goals and focus.

The "EARLY-STAGE CLINICAL phase," which usually includes Phase I and Phase II, is the first clinical trials conducted in humans after promising results have been obtained in preclinical tests and animal studies. [3]

These are two experimental drugs that are in this phase.

NTLA-2001 Transthyretin (ATTR) Amyloidosis

Initial conclusions show "NTLA-2001 was associated with only mild adverse events and led to decreases in serum TTR protein concentrations through targeted knockout of TTR [4].

2 . NTLA-2002 Hereditary Angioedema

Overall, the interim results of the study show promising progress in the development of NTLA-2002 for the treatment of HAE. The drug appears to be effective in reducing plasma kallikrein levels and HAE attack rates and is well tolerated. The data are promising and suggest that NTLA-2002 has the potential to transform the treatment of HAE and provide patients with a functional cure. This is an important step toward the application of CRISPR technology to treat genetic diseases. [5]

So back to the question, what's special about it now?

There are only a few companies that are so far with research in this area and are already testing solutions "drugs" on people.

I think it is one of the great hopes of people affected by a rare disease to be completely cured.

Intellia is thus a pioneer of a new technology that could disrupt our understanding of the world.

In this regard, I can recommend the book "The Code Breaker". It shows the development , the chances and the potentials of CRISPR-Cas9.

The author addresses the following potentials:

• Cure of genetic diseases
• Cancer treatment
• Agricultural and food industry

In addition, ethical questions are openly posed and discussed.

I find the topic very exciting and see great potential there. How do you see the development and what do you think about the genetic modification of humans?

In my opinion, this topic will come up sooner or later. It remains to be seen how the development progresses.

I invest not only because I see the potential in Intellia, but also to stay up to date.

Sources:

[1] https://www.intelliatx.com/about/ (2023)

[2] https://www.mpg.de/11018867/crispr-cas9 (2023)

[3] https://toolbox.eupati.eu/resources/basics-of-early-clinical-development/ (2023)

[4] https://www.nejm.org/doi/full/10.1056/NEJMoa2107454

[5] https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-presents-new-interim-data-first-human

Disclaimer: I am definitely not an expert, so forgive me if some information is wrong and correct it in the comments.

Hi guys, have 1000 euros to invest (unplanned) all in ETF or gambling ->$NTLA (+2,08 %)
$PYPL (+0,71 %) ? Do you have other speculative ideas?

Hi to All,

I would like to share with you my portfolio.

In terms of structure, I can say that I am heavily invested in the healthcare sector, which is certainly due to my profession. With $VRTX (-0,51 %) and $REGN (-0,34 %) I have largely stable companies in the portfolio. In addition, there are employee shares in the portfolio, which are allocated over the years. With $AXSM (+1,29 %) and $BTAI I have two up-and-coming companies that have recently launched their first product and have an interesting pipeline for me. I have sold some shares in these companies recently to take some pressure off my portfolio, as it was very dependent on Axsome, for example. Otherwise you can still discover some exciting growth stocks ($NTLA (+2,08 %) , $EDIT (+9,37 %) , $ALEC (+2,88 %) )

At the moment, I am focusing on diversifying more in the different sectors and choosing stocks that offer more support in this time of crisis. Therefore my choice is BlackRock, Pepsi, BASF, BHP and some others. (Even if BASF is stumbling a bit at the moment) In parallel, some stocks with dividends, which are always reinvested.

I am aware that my portfolio is not yet ideally structured, but I am currently working on it. Focus at the moment is on $PEP (+0,17 %)
$ZTS (+1,8 %)
$BLK . On my watchlist I have at the moment $DHR (+0,29 %) or $TMO (+0,3 %) as equipment manufacturer and distributor in the life science sector as well as $PG (+0,07 %) for the consumer staples sector.

Finally, about myself 30 years, 2 kids, living separately. Goal: A deposit for old age

Savings rate currently: 200 to 250 euros + dividends.

My personal weakness:

I find it difficult to invest in an ETF to build this as a core.

Looking forward to the feedback.

#bestworst

Since now virtually everyone posts what his best and worst investments were I will not hold back.

My #bestes investment was Intellia Therapeutics $NTLA (+2,08 %) . Bought at 60.52 € and sold at 119.90 €. Were a whole 97% return!

My #worst Investment was Beyond Meat $BYND (-3,19 %) with minus -19,94%. I find their mission very nice and would find it very very good if the prevail. Unfortunately, they have not (yet) made it. Meanwhile, there are also so many alternatives and I wonder if they can become really big again. I would wish it in any case.

In general, I have also seen with most others that the losses are limited. Getquin users apparently know what they are doing 💪