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An ancient RNA-guided system could simplify delivery of gene editing therapies By Corie Lok February 27, 2025 Breadcrumb Home An ancient RNA-guided system could simplify delivery of gene editing therapies The programmable proteins are compact, modular, and can be directed to modify DNA in human cells.
Novartis Finalizes Acquisition of Regulus Therapeutics, Strengthening Its Renal Disease Portfolio with Promising ADPKD Therapy Novartis AG, a leading global pharmaceutical company, has officially completed its acquisition of Regulus Therapeutics Inc. , a biotechnology firm known for its expertise in microRNA-targeting therapies.
Functional analysis based on these structures also revealed how a 'prime editor' could achieve reverse transcription, synthesizing DNA from RNA, without 'cutting' both strands of the double helix. Clarifying these molecular mechanisms contributes greatly to designing gene-editing tools accurate enough for gene therapy treatments.
With new modalities such as gene and cell therapies, RNA therapeutics, complex biologics and more, today’s science brings unprecedented opportunities to address diseases that have long remained out of reach.
Biogen Reports Promising Interim Phase 1 Results for Salanersen in Spinal Muscular Atrophy, Prepares for Registrational Trials Biogen has announced encouraging topline results from its Phase 1 clinical trial evaluating salanersen (BIIB115/ION306) , an investigational antisense oligonucleotide (ASO) therapy for spinal muscular atrophy (SMA).
If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant.
Altasciences at ASGCT 2025: An Open Forum for All Things Gene and Cell Therapy | By Kaylyn Koenig aasimakopoulos Thu, 07/10/2025 - 08:00 I recently returned from attending the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting.
The dynamic behaviour of RNA transcripts, the regulation and modification of proteins, the lipid composition of membranes, the structural organisation of cells and the spatial context of tissues all play critical roles in disease progression and therapeutic response.
In this article Drug Target Review’s Izzy Wood spoke to Sam Hasson, Director of Target Biology at Rgenta Therapeutics, a biotech firm in Massachusetts, US, that aims to develop small molecule therapeutics to target RNA processing. The field is faced with a number of obstacles that require specifically assay technologies to surmount.
By Allessandra DiCorato May 16, 2024 Credit: Deverman lab Brain vasculature (in blue) surrounded by RNA (in orange) transcribed from the gene delivered to the brain in humanized mice using an engineered AAV targeting the human transferrin receptor.
As our understanding of the underlying biology of disease grows more sophisticated, emerging therapies operate on increasingly complex biopathological systems and mechanisms. These fields explore highly precise biological processes related to RNA, specific proteins and gene expression mechanisms.
Founding Vision: Harnessing RNA Editing for Transformative Medicine Korro Bio’s story began in 2018, co-founded by a remarkable group: Jean-François Formela, M.D. Academic specializing in RNA editing). Their collective expertise—spanning gene editing, venture creation, and foundational science—set the stage for building Korro.
From maths and physics to molecular biology Schnall-Levin did not begin in the life sciences. “I I took a couple of other software startup opportunities,” he adds, “but I realised I missed the genomics and science. I did physics and math as an undergraduate,” he explains. I started looking around and getting interested in biology.
This includes verifying the experimental design and understanding how the data was generated whether it was from RNA sequencing, mass spectrometry, or other biological assays. About the authors Dr Raminderpal Singh Dr Raminderpal Singh is a recognised visionary in the implementation of AI across technology and science-focused industries.
This second ELRIG meeting on Therapeutic Oligonucleotides brings together esteemed scientists from academia, industry, and other members of the drug discovery community to explore the discovery, validation, and targeting of oligonucleotide-based drug candidates, including antisense oligonucleotides (ASOs) and small interfering RNA (siRNA).
“I was increasingly drawn to understanding the underlying biology of disease at a molecular level – the ‘why’ – and how we might harness science to create better medicines for patients,” Lepore says. From theory to therapy This approach is already delivering promising insights across key therapeutic areas. .
One company leading the charge in this revolution is Alltrna , whose pioneering work in engineered transfer RNA (tRNA) therapeutics is offering new hope for patients with rare genetic diseases.
Pushkal Garg to Lead Unified Research and Development Organization, Signaling Strategic Expansion into Next Phase of RNAi Therapeutics Innovation Alnylam Pharmaceuticals , a global leader in RNA interference (RNAi) therapeutics, has announced the promotion of Pushkal Garg, M.D.,
Since their first approvals in 2017, autologous CAR T-cell therapies have revolutionised the treatment of blood cancers, offering hope where previous options had failed. Consequently, these therapies come with significant burdens and are often underutilised despite their transformative patient outcomes.
Now the researchers describe how they re-engineered both eVLPs and parts of the prime editing protein and RNA machinery to boost editing efficiency up to 170 times in human cells compared to the previous eVLPs that deliver base editors. DOI: 10.1038/s41587-023-02078-y Tags: Liu Lab Gene editing Rare Disease Gene-based therapy delivery
By Leah Eisenstadt, Broad Communications October 23, 2024 Credit: Courtesy of the Broadbent family Brian and Julia Broadbent are raising their daughters Claire, top left, and Emma, seated, who is the first person to be diagnosed with a rare genetic disorder caused by the long noncoding RNA CHASERR.
I’m so proud of this team and the partnership we formed to follow the science and set up some major positive impacts on women’s health,” Blainey said. Using RNA sequencing and working with the Broad’s Metabolomics Platform and collaborators at St. Paper cited Zhu M et al.
This included compiling RNA data, as well as other data points such as patient demographics, clinical treatment arm and patient diagnosis. However, for others, it did not work – or did so only marginally, or with serious side effects,” said Stuart Henderson, global Life Sciences lead for Accenture.
Novartis today announced a license option agreement with Voyager Therapeutics, a gene therapy company focused on next-generation adeno-associated virus (AAV) technologies, for three capsids to use in potential gene therapies for neurological diseases, with options to access capsids for two other targets. About Novartis.
7 In immune therapies, MSI helps monitor immune-cell distribution and off-target effects, optimising therapeutic strategies and improving patient outcomes. Stefan has held pivotal roles at leading life science companies including Roche and Siemens Healthineers.
Most targeted cancer therapies used today operate by inhibiting targets along well-known oncogenic signalling cascades. The reactivation of oncogenic signalling upstream or downstream of the driving oncogene is a well-studied source of resistance to targeted cancer therapies.
KIF1A in New York: The 2023 KAND Family & Scientific Engagement Conference We won’t be posting next Saturday, but not for a lack of science! Genetic and RNA-based therapies seek to correct mutant KIF1A – improving its force generation, velocity, and distance traveled by increasing the ratio of healthy to mutant KIF1A.
Daily Dive M-F Commercialization Weekly Every Wednesday Gene Therapy Weekly Every Thursday Emerging Biotech Weekly Every Tuesday By signing up to receive our newsletter, you agree to our Terms of Use and Privacy Policy. Nolan Townsend said will help “realize the therapeutic and economic value of promising preclinical science.”
FDA classifies it as a “nonsteroidal treatment” – not a gene therapy, but it affects gene expression. But even if gene therapy could reach sufficient muscle cells to improve quality of life, another hurdle looms: the dystrophin gene that’s deleted in DMD is huge, the largest in a human genome at 2.2
Over the past two decades, life sciences venture capital has developed a substantial body of data, pattern recognition, and tacit knowledge around what works in early-stage biotech. The structures that support them, how we align, coordinate, and decide, may need to evolve as much as the science they advance.
Norbert Makori and Pierre Jolicoeur: The Benefits of Combining Preclinical and Bioanalytical for Your Gene Therapy Studies pmjackson Fri, 03/01/2024 - 22:20 Recent advances in gene therapy have allowed for diseases to be approached differently. q: How can Altasciences support sponsors’ Gene Therapy Programs?
Mount Sinai researchers have developed a new model that uses DNA and RNA sequencing data from hundreds of patients to identify specific genes and genetic alterations responsible for never-before-defined subtypes of a blood cancer called multiple myeloma. doi: 10.1126/sciadv.abg9551. Source link: [link].
Making a little tangle of DNA visible to the eye and understanding that this is the basis of complex organisms, which might be altered in disease, was the defining moment for my future path in life sciences. Being an only child of non-academic parents, I learned to work and study independently early on.
I studied Natural Sciences at the University of Cambridge, where I was exposed to a range of disciplines from chemistry to physiology, specialising in my last year in biochemistry. Pushing the boundaries of science always opens the door to new, impactful developments, although there are always challenges along the way.
In the midst of the global pandemic, the analysis found no antiretroviral therapy interruptions across the entirety of the ongoing clinical development programme for long-acting cabotegravir and rilpivirine. Of those participants who transitioned back to injectables, the median duration of oral therapy was 51 days.
The studies are from groups at the Broad, Allen Institute for Brain Science, the Salk Institute for Biological Studies, and other institutions that are part of the National Institutes of Health’s Brain Research Through Advancing Innovative Neurotechnologies ® Initiative, or The BRAIN Initiative — Cell Census Network ( BICCN ). and Virginia W.
They had the science and the technology but not the business model. Researchers were able to use our synthetic RNA controls as a reference to verify and validate assays. Often, DNA technology is used in the development of new therapies. Twist was officially founded in 2013. At that time synesthetic biology wasn’t cool.
These are incredible forecasts and CAGRs, which if we assume directionally correct, rely not only on steady growth for approved therapies but also a substantial success rate of, and continued investment in, the development pipeline. TfR1 is almost becoming table stakes for Pharma and biotech looking to deliver to the brain and / or muscle.
This was a great gig – good people, good science. However, in June I happened to attend the Gordon Research Conference on Chemical Biology and High-throughput Chemistry where I saw a session on small molecules and RNA. Making small-molecule drugs against RNA structures looked like a really cool problem – I want to do THAT!
December is bringing frost in the North and plenty of cold hard cash for these life sciences companies. . European venture capital firm Forbion rounded up $545 million for its fifth life sciences fund. BioAge is on the cusp of taking pilot therapies BGE-117 and BGE-175 into clinical trials, targeting the first half of 2021.
This method was more about serendipity than science. Another promising avenue is the use of technologies like RNA interference and gene editing, which allow scientists to turn off the production of certain proteins altogether. But as molecular biology has advanced, so too has our approach to finding new drugs.
20, 2020 /PRNewswire/ — Caris Life Sciences® , a leading innovator in molecular science focused on fulfilling the promise of precision medicine, announced today that Winship Cancer Institute of Emory University (Winship) has joined Caris’ Precision Oncology Alliance (The Alliance/POA). . IRVING, Texas and ATLANTA , Oct.
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