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Showing posts with label Crispr. Show all posts
Showing posts with label Crispr. Show all posts

Saturday, January 18, 2025

Cutting-Edge Healthcare Advancements: 2025 Outlook

 


This report highlights eleven publicly traded companies at the forefront of medical innovation. Each organization is driving progress through breakthrough technologies—ranging from gene editing to advanced surgical systems—and is poised to enhance healthcare outcomes by 2025. While each company focuses on different aspects of healthcare, they share a common theme: leveraging innovation to improve patient care, reduce disease burden, and transform standards of practice.


1. Moderna (NASDAQ: MRNA)

Primary Focus: mRNA Vaccines & Therapeutics
Key Advancements:

  • mRNA Platform Expansion: Known for its COVID-19 vaccine, Moderna is expanding mRNA-based treatments for cancer, rare genetic disorders, and various infectious diseases (e.g., influenza, RSV).
  • Personalized Cancer Vaccines: Working on individualized cancer vaccines that prime the immune system to target tumor-specific antigens.
  • 2025 Outlook:
    • Faster Vaccine Development Cycles: The proven modularity of mRNA platforms could enable rapid updates in response to emerging pathogens.
    • Broader Therapeutic Pipeline: If pivotal trials succeed, mRNA therapies may offer new standards of care for conditions lacking effective treatments.

2. CRISPR Therapeutics (NASDAQ: CRSP)

Primary Focus: Gene Editing (CRISPR-Cas9)
Key Advancements:

  • Ex Vivo Gene Editing: Lead programs focus on correcting genetic mutations for beta thalassemia and sickle cell disease by editing patients’ cells outside the body.
  • Oncology Pipeline: Investigating CRISPR-based therapies to engineer immune cells (e.g., CAR-T cells) that more effectively target cancers.
  • 2025 Outlook:
    • Potential Curative Treatments: Pending regulatory approval, CRISPR-based therapies may become one-time cures for hereditary blood disorders.
    • Scalability & Access: Demonstrated success in initial indications may pave the way for broader use in other genetic conditions.

3. Editas Medicine (NASDAQ: EDIT)

Primary Focus: Gene Editing (CRISPR & Base Editing)
Key Advancements:

  • In Vivo and Ex Vivo Editing: Developing CRISPR-based therapies for ocular diseases (e.g., Leber Congenital Amaurosis) and other rare genetic conditions.
  • Base Editing Exploration: Investigating base-editing tools for precise, single-nucleotide modifications without creating double-stranded breaks.
  • 2025 Outlook:
    • First-in-Human Ocular Gene Editing: Editas’s trials in vision restoration could transform the approach to inherited retinal diseases.
    • Expanding Applications: If initial programs prove safe and efficacious, base editing may target a wide range of genetic mutations with fewer off-target effects.

4. Intellia Therapeutics (NASDAQ: NTLA)

Primary Focus: In Vivo Gene Editing
Key Advancements:

  • Transthyretin Amyloidosis (ATTR) Program: Showcased successful in vivo gene editing by delivering CRISPR directly to the liver.
  • Broader Pipeline: Targeting other hereditary conditions such as hereditary angioedema and exploring oncology applications.
  • 2025 Outlook:
    • Validated In Vivo Approach: Demonstrated clinical proof-of-concept could open doors to editing diseases at the source.
    • Streamlined Therapies: Potential single-dose treatments for chronic, life-limiting conditions, significantly improving patients’ quality of life.

5. Beam Therapeutics (NASDAQ: BEAM)

Primary Focus: Base Editing
Key Advancements:

  • Precise Single-Base Changes: Beam’s platform edits just one “letter” of DNA, reducing the risk of large-scale genomic disruptions.
  • Broad Pipeline Targets: Includes programs for cardiovascular, blood, liver, and ocular disorders, among others.
  • 2025 Outlook:
    • Reduced Off-Target Effects: Base editing may offer safer alternatives to standard CRISPR approaches, which can cause double-stranded breaks.
    • Accelerated Clinical Trials: If Beam’s initial clinical programs show strong safety and efficacy, the technology could rapidly expand to treat a wide variety of monogenic disorders.

6. Illumina (NASDAQ: ILMN)

Primary Focus: Next-Generation Sequencing (NGS)
Key Advancements:

  • High-Throughput Genome Sequencing: Illumina’s platforms power large-scale genetic research and clinical diagnostics.
  • Cost Reduction & Accessibility: Continually driving down the cost per genome, making personalized medicine more feasible.
  • 2025 Outlook:
    • Precision Medicine Standard: Routine genomic profiling in cancer, rare diseases, and newborn screening could become standard of care.
    • Population Genomics: Wider adoption in population health studies and preventive care, enabling earlier detection of risk factors and targeted interventions.

7. Intuitive Surgical (NASDAQ: ISRG)

Primary Focus: Robotic-Assisted Surgery
Key Advancements:

  • da Vinci Surgical System: A robotic platform enabling greater surgical precision and minimal invasiveness.
  • Expanded Surgical Applications: Ongoing R&D to extend robotic surgery into more complex procedures (e.g., thoracic, bariatric, and cardiac surgeries).
  • 2025 Outlook:
    • Standardization of Robotic Surgery: As more hospitals adopt these systems, patients could experience fewer complications and shorter recovery times.
    • Improved Surgeon Training & Efficiency: Next-generation technologies, including virtual reality simulations, will further refine surgical outcomes.

8. Boston Scientific (NYSE: BSX)

Primary Focus: Medical Devices & Interventional Therapies
Key Advancements:

  • Cardiovascular Innovations: Advanced stents, ablation catheters, and heart failure devices.
  • Neuromodulation & Urology: Devices like spinal cord stimulators, deep brain stimulation, and minimally invasive urology solutions.
  • 2025 Outlook:
    • AI & Diagnostics Integration: Incorporating data analytics and AI into device monitoring to offer more personalized patient care.
    • Less Invasive Procedures: Continued shift from open surgeries to minimally invasive interventions, reducing hospital stays and improving outcomes.

9. Dexcom (NASDAQ: DXCM)

Primary Focus: Continuous Glucose Monitoring (CGM) Systems
Key Advancements:

  • Real-Time Glucose Tracking: Sensors that automatically measure and transmit glucose data every few minutes.
  • Data Integration: Partnerships with insulin pump manufacturers and smartphone apps for closed-loop insulin delivery.
  • 2025 Outlook:
    • Closed-Loop Systems: Move toward “artificial pancreas” solutions where insulin delivery is automated, significantly reducing diabetes complications.
    • Improved Accuracy & Comfort: Next-generation CGMs may have longer wear times and less invasive sensor designs, further enhancing adoption.

10. Medtronic (NYSE: MDT)

Primary Focus: Broad Medical Devices & Connected Health
Key Advancements:

  • Cardiac & Vascular Devices: State-of-the-art pacemakers, defibrillators, and heart-valve repair systems.
  • Diabetes Management: Insulin pumps and continuous glucose monitoring integrations.
  • AI-Driven Platforms: Remote monitoring solutions and predictive analytics to manage chronic conditions.
  • 2025 Outlook:
    • Personalized Medicine at Scale: Leveraging data from implanted devices to tailor therapies in real time.
    • Reducing Readmissions: Better remote monitoring can catch complications earlier, lowering costs and improving patient outcomes.

11. Edwards Lifesciences (NYSE: EW)

Primary Focus: Structural Heart & Critical Care Monitoring
Key Advancements:

  • Transcatheter Aortic Valve Replacement (TAVR): Minimally invasive heart valve therapies that reduce surgical risk.
  • Critical Care Monitoring: Advanced hemodynamic monitoring systems guiding clinical decision-making in ICU settings.
  • 2025 Outlook:
    • Mainstream TAVR Adoption: As the technology evolves, more patient populations (including lower-risk groups) may qualify for transcatheter procedures, reducing the need for open heart surgery.
    • Improved Post-Operative Outcomes: Next-generation devices and monitoring capabilities will further enhance patient recovery and long-term cardiac health.

Conclusion

These eleven companies are leading a transformative era in healthcare. Collectively, they are:

  • Rewriting the Genetic Code: Gene editing and base editing promise curative approaches for diseases once considered incurable.
  • Reinventing Treatment Modalities: mRNA vaccines, targeted therapies, and minimally invasive surgical devices are refining standards of care.
  • Elevating Patient Monitoring & Management: Continuous glucose monitoring, AI-driven diagnostics, and remote device integrations enable proactive, personalized care.
  • Driving Down Costs & Hospitalizations: Less invasive procedures, early detection, and potential “one-time” curative treatments can significantly reduce healthcare expenditures over time.

By 2025, the interplay of these innovations—and the data they generate—has the potential to significantly enhance patient outcomes, streamline clinical workflows, and move us closer to a future where healthcare is predictive, preventive, and precisely tailored to individual patient needs.

ED Notes:

We have invested in 3 of these companies and placed the others on our watch list!

Royalty Pharma (NASDAQ: RPRX) is our Healthcare "Anchor Stock"!

Sunday, July 7, 2024

Editas Medicine $EDIT has pioneered the use of CRISPR technology to develop innovative genomic medicines, potentially transforming the treatment of a wide range of genetic diseases

 


Editas Medicine is a viable player in the biotech field due to several factors:

  1. Technological Advancements:

    • Editas focuses on CRISPR-Cas9 and CRISPR-Cas12a (Cpf1) gene editing technologies, which are at the forefront of genetic engineering and therapeutic development. These technologies allow for precise, targeted modifications to the genome, opening the door to treatments for a variety of genetic diseases​ (Editas Medicine)​​ (Editas Medicine)​.
  2. Pipeline and Clinical Trials:

    • Editas has a diverse pipeline with multiple programs in various stages of development. Notably, their BRILLIANCE trial for treating Leber congenital amaurosis (LCA10), a rare genetic eye disorder, has shown promising results. The success of this and other trials will be critical for their future viability​ (markets.businessinsider.com)​​ (SciTechDaily)​.


  3. Strategic Focus:

    • The company's focus on both in vivo (within the body) and ex vivo (outside the body) gene editing expands its potential applications. This dual approach enables them to target a wide range of diseases and patient populations, increasing their market potential​ (Editas Medicine)​​ (markets.businessinsider.com)​.
  4. Partnerships and Collaborations:

    • Collaborations with other biotech firms and research institutions bolster their research capabilities and expand their technological reach. Partnerships are essential for sharing expertise, resources, and accelerating development timelines​ (markets.businessinsider.com)​.
  5. Financial Position:

    • While the company has experienced fluctuations in stock prices, ongoing support from investors and strategic financial management are vital. Recent buy ratings from analysts indicate a positive outlook based on the company's strategic focus and upcoming clinical data​ (markets.businessinsider.com)​.
  6. Regulatory and Market Challenges:

    • Like all biotech firms, Editas faces regulatory hurdles and market competition. Successful navigation of these challenges will be essential for their continued viability. Positive trial outcomes and regulatory approvals will be critical milestones​ (markets.businessinsider.com)​​ (SciTechDaily)​.

In summary, Editas Medicine's focus on cutting-edge CRISPR technology, a robust and diverse pipeline, strategic collaborations, and positive analyst ratings support its viability going forward. However, their success will hinge on the continued advancement of their clinical programs, regulatory approvals, and the ability to maintain financial stability.

The founders of Editas Medicine are notable scientists with significant contributions to the field of gene editing:

  1. Feng Zhang:

    • Feng Zhang is a prominent figure in the development of CRISPR technology. He is a core member of the Broad Institute of MIT and Harvard and has been instrumental in advancing the use of CRISPR-Cas9 for genome editing in mammalian cells. His work has been widely recognized and has earned him numerous awards, including the prestigious Canada Gairdner International Award​ (Editas Medicine)​​ (SciTechDaily)​.
  2. Jennifer A. Doudna:


    • Jennifer Doudna is a professor of chemistry and molecular and cell biology at the University of California, Berkeley. She is a co-recipient of the Nobel Prize in Chemistry in 2020 for her role in the development of CRISPR-Cas9, alongside Emmanuelle Charpentier. Her research has been pivotal in the field of gene editing, and she has received many other accolades, including the Breakthrough Prize in Life Sciences​ (Editas Medicine)​​ (SciTechDaily)​.
  3. George Church:

    • George Church is a professor of genetics at Harvard Medical School and a professor of health sciences and technology at Harvard and MIT. He has made significant contributions to various biotechnological fields, including the Human Genome Project. Church is known for his pioneering work in the development of genome sequencing and editing technologies​ (Editas Medicine)​​ (SciTechDaily)​.
  4. J. Keith Joung:

    • J. Keith Joung is a pathologist and molecular biologist at Massachusetts General Hospital and Harvard Medical School. He is recognized for his contributions to the development of gene editing technologies, including zinc-finger nucleases and CRISPR-Cas systems. Joung's work has significantly impacted the field of genome editing and its applications in medicine (Editas Medicine)​.
  5. David R. Liu:

    • David Liu is a professor at Harvard University and a member of the Broad Institute. He has developed several innovative technologies in the field of gene editing, including base editing and prime editing. Liu's research aims to create more precise and efficient methods for editing DNA, with the potential for wide-ranging therapeutic applications​ (Editas Medicine)​​ (SciTechDaily)​.

These founders have played critical roles in the development and advancement of gene editing technologies, making Editas Medicine a leading company in the biotech industry.

The top five institutional investors in Editas Medicine, Inc. are:

  1. Vanguard Group Inc. - Holds approximately 8.46 million shares.
  2. BlackRock Inc. - Owns about 7.84 million shares.
  3. Deep Track Capital, LP - Holds around 5.47 million shares.
  4. State Street Corp - Owns approximately 5.07 million shares.
  5. SPDR S&P Biotech ETF - Holds around 3.18 million shares​ (Fintel - Financial Intelligence)​​ (Stock Analysis)​.

These institutions collectively own a significant portion of the company's shares,

 reflecting strong institutional interest in Editas Medicine.

"The rapid and complete resolution of aggressive MSS colorectal cancer tumors observed in this study is unprecedented in the field" says the author Dr. Kasi



Tuesday, June 25, 2024

Going forward, how viable is Beam Therapeutics in the BioTech market?

 




Fr: ChatGPT

Beam Therapeutics appears to be a viable biotechnology company, with several strengths that contribute to its potential:

  1. Financial Stability and Future Prospects: Beam Therapeutics has demonstrated strong financial management with a substantial increase in cash reserves, which were reported to be $1.2 billion at the end of 2023. This financial position is expected to fund the company's operations into 2027, showing a robust capability to sustain its long-term research and development plans​ (Beam Therapeutics)​.

  2. Advanced Gene Editing Technology: Beam specializes in base editing technology, which allows for precise, predictable, and efficient changes at specific DNA sequences without making double-stranded breaks. This technology has broad applications in developing genetic medicines for a variety of diseases​ (Beam Therapeutics)​​ (markets.businessinsider.com)​.

  3. Strategic Partnerships and Pipeline Development: The company has formed strategic alliances, such as the significant partnership with Eli Lilly, which includes substantial financial backing and collaborative opportunities in cardiovascular disease therapies. This alliance alone could bring up to $600 million in future development payments to Beam​ (markets.businessinsider.com)​.

  4. Clinical Trials and Research Focus: Beam is actively advancing multiple clinical trials, including a Phase 1/2 trial for a CAR-T therapy candidate and other in vivo gene editing programs targeting both rare genetic and more common disorders. This active pipeline underlines the company's commitment to bringing new therapies to market​ (markets.businessinsider.com)​​ (Beam Therapeutics)​.

  5. Manufacturing and Operational Expansion: The company is also expanding its manufacturing capabilities, having initiated GMP operations in its North Carolina facility, which supports both current and future therapy production needs​ (Beam Therapeutics)​.

  6. Institutional Investors:

    Strong institutional backing indicates a high level of confidence in Beam Therapeutics' future potential and stability, reflecting its position in the biotechnology and genetic editing fields.

  7. Ownership: Beam Therapeutics is heavily supported by institutional investors, with about 99.68% of its stock owned by these entities. Some of the largest institutional shareholders include FMR LLC, Vanguard Group Inc., BlackRock Inc., and State Street Corp. Notably, FMR LLC holds a significant portion of the shares, representing a substantial stake in the company. Other major investors include Ark Investment Management LLC and Farallon Capital Management LLC, highlighting a robust interest from high-profile investment firms​ (MarketBeat)​​ (WallStreetZen)​.

Competition:

Beam Therapeutics is positioned in a competitive field of biotechnology companies focusing on precision genetic medicines. 

Its main competitors include Kymera Therapeutics, Intellia Therapeutics, Replimune Group, Editas Medicine, Sangamo Therapeutics, Repligen, Exelixis, Halozyme Therapeutics, Revolution Medicines, and ImmunityBio. These companies are all part of the medical sector, focusing on similar technologies and therapeutic approaches​ (MarketBeat)​.

In terms of financial performance and stock metrics, Beam Therapeutics stands out with a higher revenue compared to many of its peers, but also exhibits higher volatility in its stock price. Notably, Beam has a significant institutional ownership, which suggests confidence from large investors about its future growth prospects. However, it faces strong competition from companies like Kymera Therapeutics, which has been rated slightly higher in terms of analyst consensus and market sentiment​ (MarketBeat)​.

Beam Therapeutics uses advanced CRISPR-based gene editing technology, which positions it at the forefront of gene editing innovation. Despite its strong technological base and institutional backing, the company's stock trades at a relatively lower price-to-earnings ratio compared to Kymera, indicating potential undervaluation or differing market perceptions about its growth potential 

Given these factors, Beam Therapeutics shows significant promise and viability as a biotech company focused on developing precision genetic medicines. However, it's essential for investors to consider the inherent risks associated with the biotech industry, including regulatory hurdles and the technical challenges of developing new medical treatments.

Pfizer is actively using artificial intelligence (AI) and machine learning (ML) to enhance its drug development processes.





Saturday, June 22, 2024

The technologies of, Robotics, Artificial Intelligence (AI), Quantum Computing, CRISPR, DNA editing, and RNA editing—each have the potential to significantly impact economies in different ways

 Here's an analysis of their potential economic impacts and which combination might have the greatest overall effect:

  1. Artificial Intelligence (AI):

    • Economic Impact: AI can drive efficiency, innovation, and productivity across industries. It can optimize supply chains, enhance customer experiences, and create new business models. AI-driven automation can lead to significant cost savings and increased output.
    • Potential: AI is expected to add trillions of dollars to the global economy by enabling smarter decision-making and unlocking new economic opportunities.
  2. Robotics:

    • Economic Impact: Robotics enhances manufacturing, healthcare, agriculture, and service industries through automation. Robots can perform repetitive tasks with high precision, reducing labor costs and increasing productivity.
    • Potential: The adoption of robotics can lead to the reshoring of manufacturing jobs, improve operational efficiency, and support advanced manufacturing techniques.
  3. Quantum Computing:

    • Economic Impact: Quantum computing can revolutionize fields such as cryptography, materials science, and pharmaceuticals. It can solve complex problems that are currently intractable for classical computers, leading to breakthroughs in various industries.
    • Potential: While still in its early stages, quantum computing holds the potential to disrupt industries by providing unparalleled computational power for optimization and simulation tasks.
  4. CRISPR and Genetic Editing (DNA and RNA editing):

    • Economic Impact: Genetic editing technologies can transform healthcare by enabling precise genetic modifications. They can lead to the development of new therapies, improve crop yields in agriculture, and support bioengineering.
    • Potential: The economic benefits include reduced healthcare costs, improved agricultural productivity, and the creation of new biotechnological industries.

Combination of Technologies:

  • AI and Robotics: The integration of AI and robotics can lead to smart automation, where robots are not only performing tasks but also learning and optimizing processes in real-time. This combination can greatly enhance productivity in manufacturing, logistics, and service sectors.
  • AI and Quantum Computing: AI can benefit from quantum computing's ability to process vast amounts of data and perform complex calculations quickly. This synergy can accelerate advancements in AI algorithms and expand their applications.
  • CRISPR and AI: AI can assist in the analysis and interpretation of genetic data, improving the precision and efficiency of genetic editing techniques like CRISPR. This combination can drive significant advancements in personalized medicine and biotechnology.

Greatest Combined Impact: The combination of AI, Robotics, and Genetic Editing (CRISPR, DNA, and RNA editing) is likely to have the greatest overall economic impact. Here’s why:

  1. AI and Robotics: Together, they can transform manufacturing, logistics, healthcare, and many other industries by creating smart, automated systems that can learn and adapt. This can lead to unprecedented levels of productivity and innovation.

  2. AI and Genetic Editing: AI can enhance the precision and efficiency of genetic editing, leading to breakthroughs in personalized medicine, agriculture, and bioengineering. This can revolutionize healthcare and food production, significantly impacting global economies.

  3. Robotics and Genetic Editing: Robotics can automate processes in biotechnology and healthcare, such as high-throughput screening and precise surgical procedures, further enhancing the capabilities of genetic editing technologies.

While each technology individually holds significant potential, their combined application can create synergies that amplify their economic impact, driving innovation and growth across multiple sectors.


Companies combining these technologies, articles:

Pure plays in the race for Quantum Computing technology, IONQ, Quantinuum and D-wave technologies - comparisons!

Pfizer is actively using artificial intelligence (AI) and machine learning (ML) to enhance its drug development processes.

Interest in Quantum computing technology is growing. Should there be consolidation in the quantum space, one company stands out as a takeover target!



Tuesday, May 28, 2024

Ginkgo Bioworks has been bolstering its position as a first mover in Synthetic Biology!

 Here are the companies they’ve acquired and the reasons behind each acquisition:

  1. Proof Diagnostics:

    -  Ginkgo Bioworks acquired Proof Diagnostics, a Cambridge-based CRISPR test developer co-founded by Feng Zhang (an early discoverer of Crispr technology) of the Broad Institute of MIT and Harvard. 
  2. Proof Diagnostics has been working on a “future-proof” COVID-19 screener with a rapid tabletop analyzer capable of detecting not only COVID-19 but also the flu, respiratory syncytial virus, and other diseases using gene-editing technology. 
  3. Ginkgo was mainly attracted to Proof Diagnostics for its intellectual property and libraries of RNA-programmable, non-Cas enzymes called OMEGAs. These emerging OMEGAs offer potential upgrades over current genomic medicine tools, making them easier to deliver into cells. Ginkgo plans to use these enzymes to support its gene therapy services, providing an alternative approach to gene editing.
  4. Patch Biosciences: - Ginkgo acquired Patch Biosciences, a New York-based AI platform developer focused on computer-designed cargos for genetic medicines. By integrating Patch’s capabilities, libraries, and datasets, Ginkgo aims to enhance its existing genetic medicine toolkit. 

    The machine-learning models and downstream assays from Patch Biosciences will contribute to Ginkgo’s offerings.
  5. Reverie Labs:

     - Ginkgo also acquired Reverie Labs, an AI-powered drug discovery company. Reverie Labs specializes in developing biology-focused foundation models and small molecule design. Four members of Reverie’s AI team, including Chief Technology Officer Ankit Gupta, have joined Ginkgo. 

    This acquisition will accelerate Ginkgo’s work in drug discovery and strengthen its expertise in biological engineering approaches.


Additionally, it’s worth noting that Ginkgo Bioworks made its largest acquisition to date by acquiring it's competitor, Zymergen, which is expected to significantly enhance Ginkgo’s platform by integrating strong automation and software capabilities

These acquisitions demonstrate Ginkgo’s commitment to advancing synthetic biology and expanding its capabilities in molecular medicine and AI-driven research



 Ginkgo Bioworks is an American biotech company founded in 2008 by five scientists from MIT, led by Jason Kelly. 

Their specialty lies in using genetic engineering to produce bacteria with industrial applications for other biotech companies. 

Essentially, they create custom-designed microorganisms that can be used in various fields, from agriculture to pharmaceuticals.



Ginkgo Bioworks Holdings (NYSE: DNA) is owned by 57.35% institutional shareholders, 13.56% by insiders, and 29.08% retail investors.

Article:

KEY POINTS from this article:


  • 1.   The biofoundry's platform is scaling up, "big time" and customers are flocking.
  • 2.
  •        

    Powerful collaborators are signing up

    It's working with MerckModernaEli Lilly, and many others in biopharma and agriculture.

  • Ginkgo's valuation is attractive, and trading as a penny stock at all time lows!

Disclosure: we own stock in Ginkgo Bioworks ($DNA) and are accumulating!

Analysts high on Ginkgo and it's platform.

StockScan estimates an average price of $6.06 for Ginkgo Bioworks in 2025, with predictions ranging from a low of $4.78 to a high of $7.34​ (Stock Screener, Chart, and Price) This indicates a potential rise of approximately 980.45% from the last recorded price. While Ginkgo faces challenges, its long-term opportunities in #CellProgramming and #Biosecurity could drive significant growth. Keep in mind that investing involves risks, and it’s essential to conduct thorough research before making any investment decisions