Prion

Sangamo Therapeutics to Present Neurology-Focused Pre-Clinical Data From Its Epigenetic Regulation, Capsid Delivery and Genome Engineering Platforms at the 27th Annual Meeting of the American Society of Gene & Cell Therapy (ASGCT)

Retrieved on: 
Monday, April 22, 2024
Research, Infectious Diseases, Neurology, Stem Cells, Biotechnology, Health, Pharmaceutical, General Health, Science, STAC, Neuron, Central nervous system disease, Dravet syndrome, Central nervous system, MD, Sangamo Therapeutics, Cell, AAV, Tauopathy, CNS, Engineering, DNA, Charcot–Marie–Tooth disease, Patient, Gene, Society, DSM-IV codes, Prion, Angelman syndrome, ALS, Genome, Capsid, Therapy, Powder metallurgy

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the American Society of Gene & Cell Therapy (ASGCT) has accepted 20 Sangamo abstracts for presentation at the 27th ASGCT Annual Meeting being held May 7-11, 2024, in-person in Baltimore, MD and in a virtual format.
  • Presentations will focus on the progression of Sangamo’s neurology-focused pre-clinical pipeline, including data supporting innovations in zinc finger epigenetic regulation, advances in AAV capsid engineering, and discovery of next-generation integrase technology.
  • These data will showcase a novel AAV capsid, STAC-BBB, that exhibited robust and widespread central nervous system transduction in cynomolgus macaques after intravenous delivery, as well as another novel AAV capsid, STAC-150, engineered to accelerate the discovery of potent and highly specific epigenetic regulators.
  • Sangamo will also present additional data from its AAV capsid engineering platform SIFTER and its efforts for integrase evolution and utilization.

Peer-Reviewed Scientific Publication Proposes Unifying Single Toxin Theory of Brain Neurodegeneration that Identifies New Drug Targets and Treatments for Alzheimer’s Disease and Other Neurodegenerative Disorders

Retrieved on: 
Tuesday, March 26, 2024
Health, Neurology, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Toxicity, Brain, DSM-IV codes, APOE4, Patient, Biomarker, NDA, Standard of care, Prion, Doctor of Philosophy, MD, Protein, Neurotoxicity, Genotype, Treatment, Safety, ALS, Canadian International Council, Amyloid

In addition, our single toxin theory of brain neurodegeneration identifies new targets and approaches for the development of disease-modifying treatments,” said Martin Tolar, MD, PhD, Founder, President, and CEO of Alzheon.

Key Points: 
  • In addition, our single toxin theory of brain neurodegeneration identifies new targets and approaches for the development of disease-modifying treatments,” said Martin Tolar, MD, PhD, Founder, President, and CEO of Alzheon.
  • The application of biomarkers, in particular p-tau, synaptic markers, and brain volumetrics for the evaluation of disease course and therapeutic efficacy in AD trials.
  • Other neurodegenerative disorders follow the same pattern of protein dysregulation, impaired clearance, and increased aggregation, leading to neurotoxicity and loss of function.
  • In these diseases, a normal essential protein starts accumulating in the brain, misfolding and aggregating into soluble oligomers.

Sangamo Therapeutics Reports Recent Business Highlights and Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Wednesday, March 13, 2024
Health, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, STAC, Gene, Orphan drug, ALTA, Haemophilia A, BBB, Patient, Health care, Quality of life, IND, Male, ERT, Investment, Therapy, Fabry disease, Plasma, Kite, Gene expression, Partnership, Central nervous system, Novartis, CNS, Pivotal, EMA, Prion, STAAR, Enzyme replacement therapy, Biogen, Tauopathy, Workforce, BLA, AAV, Sanofi, Research, DRG, Neuron, Hematology, FDA, DSM-IV codes, Brain, Sangamo Therapeutics, MAA, Haemophilia, CTA, Mouse, Female, Traffic barrier, Sodium, MHRA, Excipient, Society, Spinal cord, Toxicology, Sigma-Aldrich, Liver, Pharmaceutical industry

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and fourth quarter and full year 2023 financial results, including meaningful data to support advancement of its neurology pipeline.
  • “In 2023, Sangamo announced the prioritization of its pipeline programs that support our focus as a neurology-focused genomic medicine company,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.
  • Revenues for the fourth quarter ended December 31, 2023 were $2.0 million, compared to $27.2 million for the same period in 2022.

Sangamo Therapeutics Announces Data From Novel Proprietary Neurotropic AAV Capsid Demonstrating Industry-leading Blood-brain Barrier Penetration and Brain Transduction in NHPs

Retrieved on: 
Wednesday, March 13, 2024
Research, Neurology, Genetics, Clinical Trials, Health, Pharmaceutical, General Health, Other Science, Science, STAC, AAV, Sangamo Therapeutics, Gene, Brain, Capsid, Gene expression, ALS, BBB, Central nervous system, Novartis, CNS, CTA, DRG, Traffic barrier, Prion, NHP, Tropism, IND, Nav1.7, Neuron, Biogen, Tauopathy, Excipient, Therapy, DSM-IV codes, Spinal cord, Liver, Animal, Vaccine

Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.

Key Points: 
  • Sangamo is exploring avenues to resume development of these programs internally, subject to receipt of adequate funding, or with new potential collaborators.
  • In NHP studies when administered intravenously at clinically relevant doses, STAC-BBB demonstrated its potential to be a leading neurotropic capsid.
  • Exhibited 700-fold higher transgene expression in neurons compared to the benchmark capsid AAV9 and outperformed all other known published neurotropic capsid variants evaluated in the study.
  • STAC-BBB was well tolerated in NHPs, with no notable treatment related pathological findings in brain, spinal cord or peripheral tissues.

Zymo Research Pioneers a Breakthrough in Large-scale Animal-free RNase A Production

Retrieved on: 
Tuesday, March 5, 2024
Ribonuclease, RNA, Prion, Research, Biotechnology, Virus, Failure, DNA, Risk, Vaccine

This breakthrough will enable the widespread use of recombinant RNase A in applications where an animal-free enzyme is preferred.

Key Points: 
  • This breakthrough will enable the widespread use of recombinant RNase A in applications where an animal-free enzyme is preferred.
  • Recognizing these challenges, Zymo Research has developed PureRec RNase A, an animal-free recombinant RNase A.
  • PureRec RNase A was developed and manufactured in Zymo Research's state-of-the-art facility in California, empowered by specialized expertise and advanced fermentation and bioprocessing technologies.
  • For more information or bulk inquiries on Zymo Research's recombinant RNase A, please check out PureRec RNase A .

Zymo Research Pioneers a Breakthrough in Large-scale Animal-free RNase A Production

Retrieved on: 
Tuesday, March 5, 2024
Ribonuclease, RNA, Prion, Research, Biotechnology, Virus, Failure, DNA, Risk, Vaccine

This breakthrough will enable the widespread use of recombinant RNase A in applications where an animal-free enzyme is preferred.

Key Points: 
  • This breakthrough will enable the widespread use of recombinant RNase A in applications where an animal-free enzyme is preferred.
  • RNase A is a critical enzyme extensively utilized in plasmid and genomic DNA purification processes due to its efficacy in degrading RNA contaminants.
  • Recognizing these challenges, Zymo Research has developed PureRec RNase A, an animal-free recombinant RNase A.
  • For more information or bulk inquiries on Zymo Research's recombinant RNase A, please check out PureRec RNase A .

Chronic wasting disease has been detected in British Columbia deer, and we need to act now

Retrieved on: 
Friday, February 16, 2024
Head, Soil, Hunting, Prion, Research, Food safety, Scrapie, Deer, CWD, Surveillance, News, Infection, Animal, Reindeer, Weight loss, Sheep, Goat, Lymph, Prevalence, Health, Culling, Water, Creutzfeldt–Jakob disease, Brain damage, Chronic wasting disease, Disease, First Nations, Movement, Transmissible spongiform encephalopathy, Tissue, Environment, Brain

Since 1996, a deadly neurodegenerative disease of cervids (deer, elk, moose, caribou, reindeer) has been spreading across Canada.

Key Points: 
  • Since 1996, a deadly neurodegenerative disease of cervids (deer, elk, moose, caribou, reindeer) has been spreading across Canada.
  • On Jan. 31, 2024, chronic wasting disease (CWD) was detected for the first time in British Columbia in two deer.

Disease-causing proteins

  • The protein is similar to other normal proteins in the body, except it’s abnormally shaped.
  • The abnormal folding of these disease-causing prion proteins — which are found most abundantly in the brain — leads to brain damage that makes the brain appear like a sponge.
  • Other TSEs include Creutzfeldt-Jakob disease in people, bovine-spongiform encephalopathy (“mad cow disease”) in cows, and scrapie in sheep and goats.
  • This means that early detection and management is critical for reducing the impact of this disease.

British Columbia’s preparations

  • has established a surveillance program to detect CWD as soon as possible.
  • Our research suggests that a robust approach to such a difficult disease will require rapid, collective and collaborative action across sectors.
  • This approach must involve wildlife managers, hunters, local communities, First Nations and researchers to integrate a number of approaches.

Surveillance and management

  • Many CWD management programs rely on removing infected animals from the landscape.
  • While it is mandatory to submit the heads from hunted cervids in select management units in B.C., in most regions, submission is voluntary.
  • Hunters can participate in CWD management and surveillance by removing the head of the animal and submitting it to a local testing station or freezer for CWD testing.
  • The public can also participate in CWD surveillance and management by reporting signs of sick animals and vehicle collisions with cervids.
  • This is why testing cervids that have been killed by vehicles is also a critical component of CWD surveillance and management.

Curbing the spread

  • CWD can spread between animals through contact with bodily fluids.
  • Legal restrictions on carcass transport and the use of urine-based scents in hunting can also reduce the unintentional spread of CWD.
  • Research has shown that community-focused communication and engagement are essential for the success of CWD management efforts.
  • In the days ahead, fostering open dialogue and collaboration will be paramount towards an effective and sustainable effort against CWD.


Kaylee Byers is the Regional Deputy Director of the British Columbia Node of the Canadian Wildlife Health Cooperative and collaborates with the Wildlife Health Program, which leads Chronic Wasting Disease surveillance in British Columbia. Sarah Robinson does not work for, consult, own shares in or receive funding from any company or organisation that would benefit from this article, and has disclosed no relevant affiliations beyond their academic appointment.

New Publication Identifies Key Proteins Involved in Amyloid Oligomer Binding and Supports Mechanism of CT1812

Retrieved on: 
Tuesday, February 6, 2024
PLOS One, SNAP, Synapse, CMO, Cognition, Microscopy, FRET, University, Protein, PLOS, Therapy, Brain, Biology, Prion, Translational Neurodegeneration, TMEM97, Doctor of Philosophy, Medical imaging

Results detected TMEM97, a protein component of the σ-2 receptor complex, in close proximity to cellular prion protein (PrPc) on Alzheimer’s brain synapses.

Key Points: 
  • Results detected TMEM97, a protein component of the σ-2 receptor complex, in close proximity to cellular prion protein (PrPc) on Alzheimer’s brain synapses.
  • These findings support the hypothesis that these receptor proteins may form a complex on the synapse surface with Aβ oligomers binding to one or both proteins.
  • “Previous work indicated that Aβ oligomers damage synapses, but until now it was not possible to know which proteins bind toxic forms of Aβ in human synapses.
  • Importantly, this FRET signal is reduced in CT1812-treated mice, suggesting that CT1812 caused the release of Aβ oligomers from their binding site and prevented them from re-binding.

Sangamo Therapeutics Announces Strategic Update and Reports Third Quarter 2023 Financial Results

Retrieved on: 
Wednesday, November 1, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Regenerative Medicine Advanced Therapy, Ethics, Therapy, Pfizer, Gene, Partnership, Head, Exposition, European Society of Gene and Cell Therapy, ALTA, AAV, Haemophilia, Hope, 65th Street Yard, Workforce, ERT, DSM-IV codes, Cell, CAR, U.S. FDA, Safety, Investment, Patient, Central nervous system, Nav1.7, MAA, 22q13 deletion syndrome, Conference, Enzyme replacement therapy, Hematology, Quality of life, Mace Head Atmospheric Research Station, Novartis, Brain, European Society for Primary Immunodeficiencies, Haemophilia A, Spectrum, FDA, IND, Fabry disease, Prion, Society, Senior, SCN2A, ESGCT, STAAR, Intellectual disability, Sclerosis, BLA, Pharmaceutical industry, Management, Vaccine, Biogen, Neuroscience, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.
  • As a result of this restructuring, Sangamo is reducing its US workforce by approximately 40%, or approximately 162 roles.
  • Sangamo expects to incur approximately $8 million-$10 million in one-time restructuring costs in the fourth quarter of 2023.
  • Revenues for the third quarter ended September 30, 2023 were $9.4 million, compared to $26.5 million for the same period in 2022.

Sangamo Therapeutics Reports Recent Business Highlights and Second Quarter 2023 Financial Results

Retrieved on: 
Tuesday, August 8, 2023
Biotechnology, Pharmaceutical, Genetics, Health, Pfizer, Central nervous system, Therapy, Gene, Haemophilia A, BLA, Biogen, Safety, Fabry disease, Phytoene synthase, Research, Brain, AAV, Science, Fast track (FDA), U.S. FDA, Sodium, Phase 3, Nav1.7, Novartis, Patient, Prion, Primate, Annual general meeting, Cell, American Society for Cell Biology, Eli Lilly and Company, MAA, FDA, STAAR, Enzyme replacement therapy, IND, Neurology, Haemophilia, Society, Pharmaceutical industry, Medical device, Vaccine, Fabry, Chroma, Sanofi, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today reported recent business highlights and second quarter 2023 financial results.
  • We are pleased to have executed several business development deals, demonstrating the excitement in Sangamo’s science and platform,” said Sandy Macrae, Chief Executive Officer of Sangamo.
  • Revenues for the second quarter ended June 30, 2023 were $6.8 million, compared to $29.4 million for the same period in 2022.
  • Additionally, revenues relating to our collaboration agreement with Kite decreased by $5.1 million, reflecting a reduction in collaboration activities during the quarter.