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    CRISPR, Gene Therapies, and Cure Pricing Conundrums

    enSeptember 17, 2022
    Who hosts the Think Fast, Talk Smart podcast?
    What does CRISPR technology allow scientists to do?
    Why is following up important in communication?
    What are some challenges faced by CRISPR innovators?
    How can persistence impact personal and professional growth?

    Podcast Summary

    • Improve communication skills with Think Fast, Talk Smart podcastLearn tips from experts on various communication aspects, including managing anxiety and being persuasive. Listen to the podcast for insights on gene editing technologies, like CRISPR, and their potential impact on agriculture and medicine.

      Effective communication skills are essential in business and life, and the Think Fast, Talk Smart podcast can help you hone these skills. Hosted by Stanford lecturer Matt Abraham, the podcast features experts sharing tips on various aspects of communication, from managing anxiety to being persuasive. With nearly 43 million downloads and the number one career podcast in over 95 countries, it's a valuable resource for anyone looking to improve their communication abilities. Another intriguing topic discussed was the exciting frontiers of gene editing technologies, specifically CRISPR. Motley Fool advisor Carl Teel explained that CRISPR can be thought of as "genetic scissors" or a "word processing editor for genes," as it allows for precise modifications to DNA sequences. The possibilities for this technology are endless, from drought-resistant crops to potential cures for genetic diseases. However, it's important to remember that innovators in this field face significant challenges, including regulatory hurdles and reimbursement issues, while followers can benefit from the groundwork laid by these pioneers. So whether you're intrigued by the potential of gene editing or looking to enhance your communication skills, there's a wealth of knowledge to be gained from the Think Fast, Talk Smart podcast and the Motley Fool's insightful discussions on emerging technologies.

    • Revolutionary Gene Editing with CRISPRCRISPR is a precise gene editing tool that offers control over editing specific DNA sequences, holds great promise for treating genetic diseases, and faces challenges in accurately inserting new genetic material.

      CRISPR is a revolutionary gene editing tool inspired by the natural defense mechanism of bacteria against viruses. Unlike traditional gene therapies that introduce genetic material into cells using viruses as vectors, CRISPR offers more precise control over editing specific DNA sequences. The CRISPR-Cas9 system, which acts like molecular scissors, can cut DNA at precise locations and potentially replace the cut with new genetic material. This makes CRISPR a promising tool for treating genetic diseases, unlike traditional gene therapies that may not integrate into the DNA or have unpredictable insertion points. The potential of CRISPR lies in its ability to edit genes precisely and permanently, making it a game-changer in the field of gene therapy. However, the challenge lies in the second part of the process - accurately inserting new genetic material at the cut site. Despite this ongoing challenge, CRISPR holds great promise for treating various genetic diseases, including those that were previously considered untreatable.

    • Revolutionizing Gene Editing with CRISPRCRISPR technology offers hope for functional cures for genetic diseases, but potential risks of irreversible changes to genetic makeup cannot be ignored.

      CRISPR technology is revolutionizing gene editing with its speed and precision, offering hope for cure and reprieve for genetic diseases like sickle cell. While the experimental data suggests functional cures for individuals, the impact on their offspring is uncertain. CRISPR is currently being explored for various diseases, including hemophilia and blindness, through both ex vivo and in vivo approaches. However, the risks of making irreversible changes to genetic makeup cannot be ignored, and safety remains a top priority. Companies like Editas Medicine and Intellia are leading the way in in vivo research, but progress is slow. Despite these risks, the potential benefits of CRISPR therapy are significant and hold great promise for the future of genetic medicine.

    • CRISPR gene editing technology's challengesDespite promising precision, CRISPR gene editing technology faces challenges including off-target effects, potential errors, germline engineering controversies, and pricing for cures

      CRISPR gene editing technology, while promising, still faces significant challenges before it can be safely and reliably used for mass treatments of less serious illnesses. The technology, which allows for precise genome editing, is not as complex as building an atomic bomb but still requires scientific knowledge and access. There are concerns about off-target effects and potential errors in inserting new genetic material. Additionally, germline engineering, which involves making changes to reproductive cells or embryos with permanent effects, is a controversial area and has already been used in a controversial case in China. Pricing cures is also a challenge, as they are not ongoing treatments. Despite these challenges, there are standards and norms in place to prevent misuse of the technology, but the potential for it to be used inappropriately cannot be ignored.

    • Shifting towards curative gene therapies and their high pricesGene therapies' high prices challenge the industry and require new pricing models, but long-term cost savings could outweigh initial investments

      The high price tags for gene therapies are a new challenge for the pharmaceutical industry, which has traditionally sold treatments for chronic conditions. The shift towards curative gene therapies, like ZINTEGLO for beta thalassemia priced at $2.8 million, is causing a significant adjustment. Companies are exploring alternative pricing models, such as paying over time or refunds if the treatment doesn't work. While these high prices might seem daunting, the argument is that the long-term cost savings could outweigh the initial investment. However, investors should be aware that these new paradigms come with challenges and uncertainties. The example of Bluebird Bio's ZINTEGLO demonstrates the potential for great science to meet real-world needs, but also the obstacles that come with it.

    • European pricing regulations challenge gene therapy companiesEurope's cost-effectiveness approach and harsh pricing negotiations impact gene therapy stock growth, making a basket investment strategy recommended for investors.

      The European government's pricing regulations have posed significant challenges for gene therapy companies, as seen with Bluebird Bio's experiences with ZINTEGLA and Skysona. Europe's cost-effectiveness approach and harsh pricing negotiations have resulted in disagreements and withdrawals of approval. For investors, a basket approach, such as investing in a genomic ETF or creating a custom basket, is a recommended strategy for the gene therapy sector. Companies like Bluebird Bio, despite successful drug approvals, have not seen significant stock growth. The CRISPR space, while promising, may not have a purely CRISPR ETF due to its small size. Serious investors should consider a basket approach to mitigate risks and capitalize on the potential growth in the gene therapy sector.

    • Challenges for innovators in revolutionary industriesInnovators in revolutionary industries face significant challenges, but if the science is sound, they're likely to find a market and succeed. Keep an eye on regulations and reimbursement developments.

      The innovator in revolutionary industries, like gene therapy and CRISPR technology, often face significant challenges due to regulatory hurdles, reimbursement issues, and the need to change established practices. Meanwhile, followers can benefit from the innovator's efforts. In the case of CRISPR Therapeutics, the low insider ownership (around 0.16%) is a common trend in biotech due to the high capital intensity of the industry and the need for numerous funding rounds. Despite this, if the science behind the potential drugs is sound, they are likely to find a market and achieve success. However, this is not a guarantee, as seen with Bluebird Bio's struggles. Overall, it's crucial to focus on the science and keep an eye on regulatory and reimbursement developments when following companies in these paradigm-shifting industries.

    • Determining ideal targets for gene editing in allogeneic CAR T drugsChallenges in identifying specific genes for editing in allogeneic CAR T drugs for cancer treatment have resulted in underwhelming data, not due to CRISPR's ineffectiveness, but because researchers are still figuring out the ideal targets to avoid immune responses.

      While CRISPR technology itself is promising, the biggest challenge lies in determining what specific genes to edit for various applications, particularly in the field of allogeneic or off-the-shelf CAR T drugs for cancer treatment. The data for these applications has been underwhelming, not due to CRISPR's ineffectiveness, but because researchers are still figuring out the ideal targets for gene editing to avoid immune responses. Beyond medicine, CRISPR holds immense potential for agriculture, resurrecting extinct species, and creating disease-resistant animals, among other possibilities. However, the practical and political feasibility of these applications is a subject of ongoing debate in a climate that is generally distrustful of science. While there may be growing acceptance for using CRISPR to treat deadly diseases, more controversial applications like human-animal hybrid organs may face greater resistance. As always, individuals should not make investment decisions based solely on podcast content, and The Motley Fool may hold formal recommendations for or against the stocks discussed.

    • Making Meaningful Connections and Following UpMaking genuine connections and following up thoughtfully can lead to opportunities, build strong relationships, and create opportunities for success. Persistence and a positive attitude are also essential in maintaining these connections and overcoming setbacks.

      The importance of making connections and following up. We talked about how every interaction, no matter how small, has the potential to lead to something meaningful. Whether it's a casual conversation with a colleague or a meeting with a potential client, making a genuine connection can lead to opportunities down the line. Moreover, following up is crucial in maintaining those connections. It shows that you value the relationship and are interested in continuing the conversation. It's not just about sending an email or making a phone call, but doing so in a thoughtful and personalized way. Another important point we touched on was the power of persistence. It's natural to face rejection or encounter setbacks, but it's essential to keep pushing forward. Every "no" brings us closer to a "yes," and each experience helps us learn and grow. Lastly, we discussed the importance of staying positive and maintaining a growth mindset. It's easy to get discouraged or feel defeated, but focusing on the progress we've made and the lessons we've learned can help us stay motivated and continue moving forward. In summary, the key takeaway from our discussion is the importance of making meaningful connections, following up, being persistent, and maintaining a positive attitude. These practices can help us build strong relationships, grow both personally and professionally, and create opportunities for success.

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