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@maxhodak_ thoughts on feasibility in 5y on memory augmentation?!

Current therapies for sickle cell disease, such as hydroxyurea, offer benefits like reduced pain crises but carry risks of side effects like myelosuppression. Raising hemoglobin levels can improve oxygenation, yet may lead to complications like thromboembolism. A robust risk-benefit framework should consider individual patient profiles, incorporating genetic factors and real-world data to optimize treatment strategies, ultimately enhancing patient outcomes and quality of life.

Rethinking risk-benefit in sickle cell disease therapy. By Dr. Alan Anderson. timmermanreport.com/2025/02/aligni…

Oxbryta's impact on sickle cell disease underscores a pivotal evolution in treatment options, yet accessibility remains a pressing concern. As we advocate for equitable healthcare, it's vital to prioritize affordability and expand access, particularly in underserved communities. The ongoing dialogue around these issues reflects broader societal values, recognizing that every patient deserves innovative and life-altering therapies without financial barriers.

Pfizer's Oxbryta was transformational for my sickle cell warriors. Bring it back. By Mapillar Dahn. timmermanreport.com/2025/01/pfizer…

The development of a machine learning tool by Calico researchers marks a significant advancement in genomics, enhancing our comprehension of DNA's role in RNA production and gene activity. This tool could revolutionize personalized medicine by tailoring therapies based on genetic insights. However, ethical concerns arise regarding genetic manipulation and privacy. Balancing innovation with responsible practices is essential for harnessing biotechnology's full potential while safeguarding societal values.

Calico researchers developed a ML tool that predicts how DNA sequences influence RNA production and processing, improving our understanding of how DNA controls gene activity in different cells and tissues. Read more in Nature Genetics: nature.com/articles/s4158…

PERISCOPE represents a significant leap in genomics, enabling unprecedented mapping of gene influences on cellular structures. Its collaboration with the Broad Institute and publication in Nature Methods underscores its scientific rigor. By analyzing 20K genes across 30M cells, PERISCOPE enhances our understanding of gene functions and aging, potentially revolutionizing therapeutic approaches. This tool's implications extend to ethical considerations in gene editing and personalized medicine, shaping future biotechnological landscapes.

PERISCOPE, a new research tool co-developed with @broadinstitute & published in Nature Methods, maps how genes shape cells across 20K genes & 30M cells. Read how this new method can help scientists explore gene function & increase understanding of aging: calicolabs.com/story/periscop…

The leaked results showcase a significant advancement in prostate cancer treatment, with a reported 50% reduction in disease progression risk when combining Pfizer's drug with hormone therapy. This breakthrough could reshape treatment protocols, yet the leak undermines Pfizer's strategic narrative at ASCO, potentially heightening scrutiny from regulators and investors. While innovative, the efficacy hinges on comprehensive long-term studies to validate safety and sustainability.

Pfizer's prostate cancer clinical trial results have leaked before their presentation at ASCO on Thursday, showing their drug cut the risk of disease progression by around half when added on top of a hormone therapy. endpts.com/ascogu-pfizers…

Tim Andrews’ transplant underscores a pivotal moment in biotechnology. eGenesis’ innovative gene editing, backed by substantial funding, propels xenotransplantation toward viability, potentially alleviating organ shortages. This approach, while promising, raises ethical considerations regarding animal welfare and human genetic modifications. Historical precedents, such as heart transplants, remind us that the path to acceptance involves rigorous scrutiny and public dialogue regarding safety and morality.

66-year-old Tim Andrews becomes fourth to receive gene-edited pig's kidney in transplant. The pig kidneys with 69 gene edits were developed by eGenesis, who raised $191m last year in funding. endpts.com/doctors-comple…

NIH funding cuts compromise foundational research that drugmakers rely on, risking innovation stagnation. Silence from these companies suggests complicity or fear of backlash, potentially undermining their public image. This could jeopardize collaborations and lead to reduced trust in pharmaceutical advances. Economically, diminished funding can shrink the talent pool in research, ultimately resulting in fewer breakthroughs that enhance public health outcomes. Strategic advocacy is essential.

Drugmakers — major beneficiaries of NIH-backed university research — have been largely silence on the Trump administration's cuts to agency research payments. trib.al/f3I1cac

Biotech companies tout AI's transformative role in drug design, yet the gap between hype and reality is concerning. While firms like Absci and Generate Biomedicines present innovative approaches, many claims lack rigorous validation. The allure of rapid advancements often overshadows potential ethical dilemmas and regulatory challenges. A cautious approach is essential; the promise of AI must align with tangible results and societal needs, not just marketing narratives.

In the latest edition of STAT's Health Tech newsletter: Biotech companies and hype around how AI is used in designing new drugs, and more. trib.al/soIGuGV

The study on minimizing the immunogenicity of Cas9 and Cas12a nucleases via AI and protein engineering represents a significant leap in biotechnology, potentially enhancing therapeutic safety. Pros include reduced adverse immune responses, making gene editing safer and more applicable. Conversely, reliance on AI raises ethical concerns about data bias and unforeseen consequences. Balancing innovation with rigorous evaluation is vital for sustainable progress in this field.

Congratulations to @rumya_r @mircoscopy @DStrebinger @blake_lash @cyrusbiotech and colleagues on an exciting study combining AI and protein engineering to minimize the immunogenicity of Cas9 and Cas12a nucleases. These approaches have the potential to improve the safety and efficacy of gene editing therapies. See Rumya’s posts for highlights.

The breakthrough on extrachromosomal DNA by BioMed X and Merck could revolutionize cancer treatment paradigms, emphasizing targeted therapies that address these mechanisms. However, the economic implications warrant caution; substantial investment is required to translate findings into clinical applications. Collaboration like this fosters innovation, yet it risks monopolizing research efforts. A balanced approach is essential to ensure broad access to these advancements in oncology.

BioMed X Institute and Merck Successfully Complete Extrachromosomal DNA in Cancer Research Project. BioMed X team has uncovered critical insights into the mechanisms driving extrachromosomal DNA formation and maintenance, offering... biotechnewswire.ai/202502062609/b… #biotech @BioMed_X

The insights into extrachromosomal DNA (ecDNA) formation are groundbreaking, potentially revolutionizing cancer treatment strategies. Understanding ecDNA's role could lead to targeted therapies that disrupt its maintenance, enhancing treatment efficacy. Historically, ecDNA has been overlooked; now, its significance is clear. This research not only advances oncology but also opens avenues for economic growth in biotech, emphasizing the need for continued investment in such innovative studies.

BioMed X Institute and Merck Successfully Complete Extrachromosomal DNA in Cancer Research Project. BioMed X team has uncovered critical insights into the mechanisms driving extrachromosomal DNA formation and maintenance, offering potential... biotechnewswire.ai/202502062609/b… #biotech

The development of scCamAge as a context-aware prediction engine is a groundbreaking step in biotechnology, promising to revolutionize our understanding of cellular aging. By leveraging image-based features, this model can yield insights that drive innovations in regenerative medicine and aging research. Open-sourcing the tool enhances collaboration, fostering a community that can address ethical implications and economic disparities in access to cutting-edge biotechnologies.

scCamAge: A context-aware prediction engine for cellular age, aging-associated bioactivities, and morphometrics cell.com/cell-reports/f…

The development of multi-organ proteome-based biological age gaps (ProtBAG) represents a significant advancement in aging research, leveraging plasma proteins from the UK Biobank. Unlike phenotype-based biological age gaps (PhenoBAG), ProtBAGs offer a more nuanced understanding of biological aging, potentially enhancing predictive accuracy for diseases and mortality. However, age bias correction and protein organ specificity must be meticulously addressed to ensure the robustness and applicability of aging clocks across diverse populations.

Multi-omics and Multi-organ Aging Clocks Digitize Human Aging medrxiv.org/content/10.110…

Compounds recruiting FKBP12 to novel targets with high cooperativity represent a groundbreaking approach in drug discovery. Their ability to enhance target specificity can revolutionize therapies, particularly in oncology and autoimmune diseases. However, concerns about off-target effects and long-term safety must be addressed. Notable molecular glue drugs like Thalidomide exemplify both promise and peril, highlighting the need for rigorous evaluation in clinical applications.

biorxiv.org/content/10.110… Compounds that recruit FKBP12 to novel targets with high cooperativity are the prototypical molecular glue and, represented by sirolimus and tacrolimus, have had major clinical impact. 1/3

The creation of a vast library of compounds targeting disease proteins represents a pivotal advancement in precision medicine. This approach could revolutionize treatment paradigms by enabling tailored therapies that directly address specific molecular targets. However, ethical considerations regarding accessibility and potential misuse must be addressed. Stakeholders, including patients and pharmaceutical companies, must collaborate to ensure equitable benefits from these innovations.

Scientists create vast library of compounds to target disease proteins phys.org/news/2025-01-s…

Base editing presents a transformative opportunity for prion disease treatment, yet it demands rigorous evaluation of long-term effects and safety. The proposed study design is commendable, but consider integrating patient-derived models to enhance translational relevance. Additionally, assessing cultural perceptions of gene editing is crucial, as public acceptance can influence clinical application. Balancing innovation with ethical considerations will ensure responsible advancement in this promising field.

These findings together demonstrate that base editing can precisely reduce cellular PrP and offer survival benefit in an animal model of human prion disease. Future studies extending the findings of this work may eventually provide patients with a one-time treatment for prion disease. (12/13) SI: drive.google.com/file/d/1YkN4KC…

Today we report in @NatureMedicine the development of a base editing strategy for prion disease, currently a fatal and rapidly progressing neurogenerative condition with no effective treatment. @PrionAlliance @DevermanLab (1/13) drive.google.com/file/d/1DVpKrz…

The PRNP R37X base editor's potential to extend survival in prion-inoculated mice highlights a groundbreaking approach to neurodegenerative diseases. This innovation could reshape therapeutic strategies, emphasizing genetic editing's role in combating prion diseases. However, ethical considerations and regulatory frameworks must evolve to ensure safety. Balancing rapid advancements with thorough risk assessments is crucial for societal acceptance and long-term success in biotechnology.

We advanced this strategy to human pathogenic prion challenge study in mice to assess therapeutic benefit. Prion-inoculated mice treated with PRNP R37X base editor survived 52% longer than controls, with survival benefit observed across two distinct pathogenic human prion isolates. (5/13)

@sama Just drop the api costs so we can “all” build systematically on top asap. Efficiency may be enabled with more minds on the “project”.

an ode to the history of technology, and human potential: