RT Nicolas Hulscher, MPH (@NicHulscher)OpenAI CEO Sam Altman and His Husband Are Funding the Creation of Genetically Engineered BabiesBy merging AI with CRISPR, they aim to “fix” humans while offshoring early embryo testing to evade oversight.First iterations of GMO children will likely endure immense suffering. ...

On the 29th of November 2018, the world woke up to a headline which sounded like science fiction. The first genetically modified humans had been born. The scientist who created them is now in prison.But the technology which made them possible has not gone away. When you hear the term genetically modified, your mind might jump to ideas of designer babies edited to be taller, prettier, cleverer. However, this was not the case here.Hajjangqui was the first scientist to use gene editing to disable the CCR5 gene ...

Summary of the Genetic Medicines Conference - Editas Medicine Industry Overview - The conference focused on the genome editing industry, highlighting advancements in technologies and programs related to gene editing therapies [1][2] - The discussion included various companies involved in the field, such as Editas Medicine, Arbor Bio, Prime Medicine, Yultec Therapeutics, and GenEdit Bio [2] Key Points and Arguments Company Introductions - **Yultec Therapeutics**: Focuses on in vivo genome editing therapies with five clinical programs, targeting diseases from ultra-rare to chronic cardiovascular conditions [4] - **Editas Medicine**: An in vivo CRISPR gene editing company with a lead program, EDIT401, showing a 90% reduction in LDL cholesterol levels, aiming for clinical trials by the end of 2026 [6] - **GenEdit Bio**: Developing affordable DNA surgeries for genetic diseases, with a focus on eye diseases and CAR T cell therapies [8][10] - **Prime Medicine**: Specializes in prime editing technology, targeting liver diseases and working on various genetic disorders [11][12] - **Arbor Bio**: Focuses on in vivo genome editing with a broad platform of editing approaches, including programs targeting the CNS [13][14] Technological Advancements - The genome editing field has expanded to include various technologies such as nuclease editing, base editing, and prime editing, with discussions on how these technologies will coexist and evolve [15][16] - The importance of product differentiation and the need for effective delivery systems were emphasized, with a consensus that delivery is as critical as the editing technology itself [28][29] Lessons Learned from Initial Therapies - The technical success rates from preclinical to clinical stages have been unprecedented, highlighting the rapid advancement of gene editing technologies [25][26] - The need for companies to focus on differentiation and the commercial viability of one-time therapies was discussed, with an emphasis on the economic arguments for such treatments [27][46] Commercial Viability and Market Considerations - The commercial case for one-time gene editing therapies was debated, with examples of partnerships and the importance of understanding the rare disease market [50][51] - The potential for gene editing therapies to transform patient care was highlighted, with a focus on the need for effective pricing strategies and understanding the current standard of care [55][61] - The discussion included the importance of low-cost delivery systems to enhance commercial viability and patient access to therapies [59][61] Future Outlook - The panelists expressed optimism about the future of gene editing, predicting multiple approvals and launches in the coming years, alongside an evolving payer landscape that may better accommodate these therapies [68] Additional Important Insights - The variability of clinical responses in edited patients is narrower compared to other therapeutic modalities, which may lead to higher responder rates and lower treatment costs [53][54] - The need for companies to adapt their business models and focus on specific commercial opportunities was emphasized, indicating a more disciplined approach in the industry [65][66] This summary encapsulates the key discussions and insights from the conference, providing a comprehensive overview of the current state and future potential of the genome editing industry.

.@Shea_ARK shares more breakthroughs on gene-editing’s journey toward curing disease.Shea Wihlborg (@Shea_ARK):Wow, exciting work out last week on a new gene editing system called bridge editing, which could expand the range of diseases addressable by genetic medicine.Where CRISPR fixes “typos,” bridge editing may be able to rewrite an entire chapter of DNA—up to nearly 1 million base ...

Crispr Technology & Applications - Crispr 基因编辑技术能够对患者的 DNA 进行精确编辑，从而识别新的治疗靶点并纠正某些疾病，扩大可治疗疾病的范围 [2] - Crispr 技术与 AI 结合，加速识别靶点和疗法，从而加快新疗法上市的速度 [3] - 癌症，尤其是无法以其他方式治疗的儿童疾病和罕见癌症，可能是 Crispr 技术首先产生影响的领域 [5] Regulatory Landscape - FDA 对新的治疗方法和技术平台持支持态度，并倾向于快速批准 [5] - 美国卫生与公众服务部 (HHS) 部长承诺支持细胞和基因治疗领域，这对行业来说是一个巨大的推动力 [7] - 众议院拨款委员会也表达了对细胞和基因治疗领域的支持，释放了积极信号 [7] Manufacturing & Supply Chain - 该公司主要在美国本土进行生产，对进口尤其是来自中国的依赖程度不高 [9] - 公司在马萨诸塞州进行生产，并在匹兹堡进行扩张 [9] - 匹兹堡的团队致力于支持当地学校的 STEM (科学、技术、工程和数学) 项目 [9]

Investment Rating - The report indicates a strong investment interest in the plant biotech sector, with a notable increase in venture capital funding and a focus on early-stage deals, despite a broader downturn in agtech funding [4][8]. Core Insights - Plant biotech startups attracted $1.2 billion in VC funding in 2024, marking a 78.3% year-over-year increase, demonstrating sector resilience [4]. - Gene-editing technologies like CRISPR have democratized seed development, allowing startups to innovate rapidly without the high costs associated with traditional GMO standards [4][10]. - The rising costs of fertilizers and pesticides are driving the adoption of biological inputs, with AI technology expected to enhance target identification and reduce development costs [4][8]. - Successful exits for agricultural biotech startups have been rare, making the next few years critical for establishing success in the plant biotech space [4]. Overview - Advances in biotechnology over the past 20 years have transformed agriculture, with genetic engineering becoming standard and biological inputs emerging as sustainable alternatives to traditional agrochemicals [7]. - The VC ecosystem has significantly contributed to the growth of plant biotech startups, with cutting-edge technologies enabling rapid innovation in agricultural sustainability and productivity [8]. Opportunities - AI and machine learning applications are revolutionizing the identification of gene targets and beneficial microbial interactions, streamlining the development of new crop varieties and biological products [36]. - Gene editing is facilitating the development of new traits at unprecedented speed and precision, with several startups approaching commercialization of innovative crop varieties [37]. Technologies and Processes - The report categorizes various segments within plant biotech, including genetic improvement, plant data and diagnostics, bionutrients, and biocontrol solutions, highlighting key startups in each area [28][30][33]. - Startups are leveraging advanced genetic technologies and bioinformatics to enhance crop traits, assess plant health, and develop sustainable agricultural practices [29][30][31]. Recent Deal Activity and Market Outlook - Despite a decline in overall agtech VC deal activity, plant biotech saw a significant increase in deal value, attributed to large rounds for maturing biotech companies and sustained interest in early-stage deals [47]. - The report notes that while VC funding for plant biotech startups is substantial, successful exits remain uncommon, with a few notable IPOs in related sectors [48].