MartinLutherKing
2 일 전
The world awaits Nobel Prize Highlights MicroRNA’s Promise in Cancer Therapy
The Nobel Prize in Medicine has been awarded to Victor Ambros and Gary Ruvkun who discovered microRNA and its role in the regulation of genes.
The two scientists discovered a new principle of gene regulation that is contributing to the development of drugs to treat cancer and heart disease, as well as neurological conditions such as Alzheimer’s disease and Parkinson’s disease.
The So What
“It’s time for major pharma companies to double down on building knowledge and capabilities, and consider acquiring licenses for drugs and clinical-stage assets that have shown promise to impact large populations,” says Jorge Vazquez Anderson, a partner at BCG who is also an RNA scientist.
“Many major pharma companies have already invested, it is time for them to do it more decisively.”
Novel therapies and medicines typically begin by tackling rare diseases, and RNA therapies have followed this pattern.
There are already existing RNA technologies that are commercially viable and in use:
The best known—messenger RNAs—were used in the COVID vaccines produced by Moderna and by Pfizer and BioNTech.
Biopharma firms such as Alnylam and Ionis are leading on small interference RNA to help cure rare hereditary diseases or genetic birth defects such as spinal muscular atrophy or amyloidosis.
The COVID vaccines helped establish broader proof of efficacy and safety, and RNA-based therapies are now starting to address diseases that impact much larger numbers of people.
MicroRNA is a subset of other RNA technologies and an example of the acceleration of the RNA innovation wave.
Clinical trials of treatments based on microRNA are already in progress for cancer, heart disease, and neurodegenerative disorders.
RNA is the ribonucleic acid present in living cells that carries instructions coded in DNA to make proteins. Until recently, it was considered a mere messenger, but now many regulatory functions have been discovered too. MicroRNAs as therapeutics agents are an example of harnessing these regulatory properties. MicroRNAs can disrupt a disease from damaging or distorting cells by binding to messenger RNAs.
“MicroRNA takes the efficacy of potential drugs to the next level by tackling multiple genes at once,” says BCG Managing Director and Partner Shana Topp who specializes in cell and gene therapy manufacturing.
“RNA also has other advantages over other gene therapies: It is simpler from a manufacturing standpoint. And, as demonstrated by the rapid development of COVID vaccines, it is easier and simpler to predict how an RNA molecule will behave. This can accelerate lead times to design and develop drugs.”
Now What
There are three key challenges when working specifically with microRNAs:
Delivery systems. Efficiently delivering microRNA-based therapies to specific tissues or cells without inadvertently affecting other areas.
Stability. MicroRNAs being used as treatment need to be stable in the body and resist enzymatic degradation. RNA-based molecules are much less stable than DNA or proteins.
Safety. Minimizing unintended interactions and immune responses is critical for clinical success. Unintended interactions are more likely because of potential binding given weaker attraction to other molecules with sequence similarities.
These challenges have two major implications for drug development and manufacturing:
Design. The function of the RNA depends on the sequencing of the units within, including the way RNA molecules fold into specific 3D structures. A deep understanding of the role of sequence and structure of the RNAs, including chemical modifications, will be needed to better control unintended interactions.
Delivery systems. There needs to be continued innovation on more efficient delivery systems such as lipid nanoparticles—layers of fat that build a protective shell around the microRNA to protect it from degradation while reaching its target. Viral vectors, which use modified viruses to deliver microRNA to cells, is another potential solution.
“Specialist firms are leading the way at present, especially when it comes to IP, talent, and development knowhow,” Topp says.
“To take advantage of this wave of innovation, industry players should develop a strategy to build the required capabilities, either through acquisitions, partnerships, or internal investments.”
https://www.bcg.com/publications/2024/nobel-prize-highlights-microrna-in-cancer-therapy
MartinLutherKing
2 일 전
Big hope for cancer: How the Nobel-winning discovery of microRNA is changing the face of diagnosis and therapies
Besides cancer, researchers across the country are working with microRNA to diagnose and treat Alzheimer’s and viral infections.
What if the ammunition to fight diseases like cancer and Alzheimer’s lies in the smallest part of your cells? We all know that DNA (deoxyribonucleic acid) contains the blueprint of life and the RNA (Ribonucleic acid) helps in transcribing the information and producing the proteins to sustain it. However, it is a tiny molecule called microRNA, which helps cells control the types and amounts of proteins they make, that has opened up new pathways for diagnosing and treating diseases — from cancers and Alzheimer’s to viral infections.
Scientists Victor Ambros and Gary Ruvkun were awarded the Nobel Prize for medicine for their discovery of microRNA in 1993. Now, researchers across India are working with these molecules to treat the untreatable and diagnose conditions in their early stages.
https://indianexpress.com/article/health-wellness/cancer-nobel-prize-microrna-alzehimers-dna-9689415/lite/
MartinLutherKing
3 일 전
E Pluribus Unum brothers The one and only
FDA Approves Phase 1/2 Trial for TransCode's TTX-MC138 in Advanced Tumor Patients
3 June 2024
TransCode Therapeutics, a company dedicated to the advancement of RNA therapeutics in oncology, has received the green light from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2 clinical trial of their lead candidate, TTX-MC138. This approval is a significant step forward for the company, as it allows them to begin testing the safety and efficacy of TTX-MC138 in patients with advanced solid tumors.
The Phase 1/2 trial is structured to first determine the safety and tolerability of TTX-MC138 through a dose-escalation phase. This will be followed by a dose-expansion phase, where the drug's safety, tolerability, and anti-tumor activity will be further assessed in patients with specific tumor types. The trial aims to include a diverse range of metastatic solid cancers, with the goal of evaluating the drug's potential to impact a broad spectrum of cancers.
TransCode Therapeutics holds a strong belief in the transformative potential of TTX-MC138. Preclinical studies in animal models have shown that the treatment can significantly reduce the metastatic burden and extend survival times compared to control treatments. The company is optimistic that TTX-MC138 could offer a new therapeutic approach for patients with metastatic cancers.
TransCode is a clinical-stage company with a focus on metastatic diseases. They are leveraging their proprietary TTX nanoparticle platform to develop RNA therapeutics that can intelligently target and effectively treat cancer. TTX-MC138 is designed to target metastatic tumors that overexpress microRNA-10b, a well-documented biomarker associated with cancer spread. The company is also working on a pipeline of innovative RNA therapeutic candidates that aim to address the challenges of RNA delivery and provide access to novel genetic targets for cancer treatment.
The FDA's clearance of the IND application is a testament to the rigorous preclinical work and the potential impact of TransCode's therapeutic approach. The Phase 1/2 clinical trial is a crucial step in the journey to bring a new treatment option to cancer patients. The company is poised to contribute to the field of oncology with its RNA-based therapeutics, offering hope for improved patient outcomes in the fight against cancer.
MartinLutherKing
3 일 전
E Pluribus Unum brothers The one and only
FDA Approves Phase 1/2 Trial for TransCode's TTX-MC138 in Advanced Tumor Patients
3 June 2024
TransCode Therapeutics, a company dedicated to the advancement of RNA therapeutics in oncology, has received the green light from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1/2 clinical trial of their lead candidate, TTX-MC138. This approval is a significant step forward for the company, as it allows them to begin testing the safety and efficacy of TTX-MC138 in patients with advanced solid tumors.
The Phase 1/2 trial is structured to first determine the safety and tolerability of TTX-MC138 through a dose-escalation phase. This will be followed by a dose-expansion phase, where the drug's safety, tolerability, and anti-tumor activity will be further assessed in patients with specific tumor types. The trial aims to include a diverse range of metastatic solid cancers, with the goal of evaluating the drug's potential to impact a broad spectrum of cancers.
TransCode Therapeutics holds a strong belief in the transformative potential of TTX-MC138. Preclinical studies in animal models have shown that the treatment can significantly reduce the metastatic burden and extend survival times compared to control treatments. The company is optimistic that TTX-MC138 could offer a new therapeutic approach for patients with metastatic cancers.
TransCode is a clinical-stage company with a focus on metastatic diseases. They are leveraging their proprietary TTX nanoparticle platform to develop RNA therapeutics that can intelligently target and effectively treat cancer. TTX-MC138 is designed to target metastatic tumors that overexpress microRNA-10b, a well-documented biomarker associated with cancer spread. The company is also working on a pipeline of innovative RNA therapeutic candidates that aim to address the challenges of RNA delivery and provide access to novel genetic targets for cancer treatment.
The FDA's clearance of the IND application is a testament to the rigorous preclinical work and the potential impact of TransCode's therapeutic approach. The Phase 1/2 clinical trial is a crucial step in the journey to bring a new treatment option to cancer patients. The company is poised to contribute to the field of oncology with its RNA-based therapeutics, offering hope for improved patient outcomes in the fight against cancer.
MartinLutherKing
3 일 전
The Overinflated Claims of mRNA Caner Cure Vaccines
The inclusion of mRNA-based cancer vaccines within the Stargate initiative raises profound concerns. Critics point to overblown claims of efficacy and safety, as well as the harrowing lessons of COVID-19 mRNA vaccines, which include growing concern they have fueled a global epidemic of so-called ‘turbo cancers’:
Unprecedented Adverse Events: COVID-19 mRNA vaccines recorded the highest rates of adverse effects, including fatalities, of any vaccine platform, according to OpenVAERS.com. Suppressed data, and government and private sector collusion to silence vaccine injury (including US Surgeon General Vivek Murthy’s vaccine injury and death coverup) stories exacerbate public mistrust.
Lack of Success in Cancer Research: Despite claims, no mRNA platform has successfully addressed cancer. Clinical trials have often fallen short of expectations, showing limited efficacy and raising concerns about unintended side effects, such as immune system overactivation or carcinogenic risks. Critics argue that the platform's inherent instability and reliance on lipid nanoparticles pose additional hurdles to safe and effective implementation.
Ethical Concerns: Coercive measures related to mRNA vaccines deployed under an Emergency Use Authorization by the Pentagon during the pandemic have severely undermined faith in public health initiatives, sparking calls for greater accountability and transparency when it comes to novel, transgenic technologies co-developed by Pharma and DARPA.
https://sayerji.substack.com/p/trumps-stargate-sparks-maha-pushback?utm_campaign=post&utm_medium=web
Money hunt
3 일 전
With TTX Ai platform, Yes RNAZ can
Can Trump-backed Stargate project beat ‘The Big C’?
The tech is expected to help create a cancer vaccine in just 48 hours.
US President Donald Trump has unveiled a $500b joint venture among Oracle Corp., OpenAI, and Softbank Group called Stargate, which promises to find a cure for cancer using the biggest artificial intelligence (AI) infrastructure.
Stargate will start building out data centres and the power infrastructure needed to further develop the fast-evolving AI in Texas, according to the White House. The initial investment is expected to be $100 billion and can reach five times that sum.
One of the initiatives to look forward to is an mRNA cancer vaccine, Oracle co-founder and Chief Technology Officer Larry Ellison told a news briefing streamed live on YouTube on Jan. 21. “Imagine early cancer detection, the development of a vaccine tailored to your specific cancer,” he said.
Ellison said the cancer vaccine could be developed robotically using AI in just 48 hours.
These vaccines use a tiny piece of mRNA, a genetic material, to teach the body’s immune system to recognise specific targets such as cancer cells, according to the Royal College of Pathologists.
“You can do early cancer detection with a blood test,” Ellison said. “Once we gene-sequence the cancer tumour, you can then design a vaccine specifically for the individual. This is the promise of AI and the future.”
While North America is boosting efforts to find a cure for cancer, the Asia-Pacific region is recognised as a key emerging player in the global AI oncology market, according to Transparency Market Research.
“Countries such as Germany, the UK, China, and India are increasing their focus on AI applications,” it said.
As of 20 August 2024, 23 cancer vaccines were in clinical trials in China, with most being therapeutic vaccines — a type of immunotherapy that treats diseases after they have occurred.
In Singapore, the National University Hospital (NUH), Thermo Fisher Scientific, Inc., and MiRXES Pte Ltd. formed a joint venture to advance early cancer detection by developing genomic solutions tailored to the Southeast Asian population.
Meanwhile, Australia is building a research centre to advance immunotherapy through real-time monitoring of patients' immune responses. The centre will integrate imaging technology with advanced tools to study cancer immunology at the cellular level.
Questions to ponder:
How can AI develop cancer treatments quickly without sacrificing patient safety?
What are the limitations of AI in cancer care, and what research is being done to address them?
What are the ethical considerations in using AI for cancer diagnosis and treatment?
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