AAV

Jaya Biosciences Presents Promising Preclinical Data in Alzheimer’s Disease at the 20th Annual WORLDSymposium™ 2024

Retrieved on: 
Monday, February 12, 2024
SAN, Toxicology, Research, APP, Plaque, PPT1, Life, Haploinsufficiency, Science Advisory Board, CNS, GUSB, Patient, GALC, Gene, IDUA, Q&A, NAGLU, IND, AAV

SOUTH SAN FRANCISCO, Calif., Feb. 12, 2024 (GLOBE NEWSWIRE) -- Jaya Biosciences, Inc. (“JayaBio” or “the Company”), a privately held early-stage life-sciences company developing CNS-directed gene therapies to address unmet needs in genetically defined neurodegeneration, reported non-clinical data at the 20th Annual WORLDSymposium™, a leading research conference on lysosomal diseases. During a late-breaking news platform presentation, Jaya Biosciences’ scientific founder, Prof. Mark Sands, reported preliminary human genetic analysis suggesting that heterozygous loss-of-function mutations in lysosomal enzyme genes are enriched in Alzheimer’s patients, as well as preclinical efficacy results in the animal model of Alzheimer’s disease (AD) for JB111, the Company’s lead therapy.

Key Points: 
  • “We are thrilled to showcase a new paradigm for the treatment of genetically defined neurodegeneration at the 20th WORLDSymposium™,” said Pawel Krysiak, President and CEO of JayaBio.
  • “The preclinical data we presented demonstrate a tremendous promise of targeting PPT1 haploinsufficiency in Alzheimer’s disease.
  • Heterozygosity of five different lysosomal enzyme genes (PPT1, NAGLU, GALC, IDUA, GUSB) significantly affects amyloid precursor protein (APP) processing and favors pro-amyloidogenic pathway.
  • CNS-directed, AAV-mediated gene therapy significantly increases the life span and improves cognitive function of 5xFAD/PPT1+/- mice.

Ruxoprubart (NM8074) Scores FDA Orphan Drug Designation for Paroxysmal Nocturnal Hemoglobinuria (PNH) Treatment

Retrieved on: 
Monday, February 12, 2024
LDH, Infection, FDA, Food, Hemoglobinuria, Paroxysmal nocturnal hemoglobinuria, Alternative complement pathway, ODD, Safety, Atypical hemolytic uremic syndrome, ANCA, Death, Patient, Hyperlipidemia, AP, Diagnosis, AAV, PNH, Chelsea Handler, Hemolysis, Pharmaceutical industry

FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.

Key Points: 
  • FDA grants orphan drug designation to Ruxoprubart (NM8074) for the treatment of PNH.
  • The FDA has approved phase 1b/Phase II clinical trials for Paroxysmal Nocturnal Hemoglobinuria (PNH), C3 Glomerulopathy (C3G), Atypical Hemolytic Uremic Syndrome (aHUS), and most recently, ANCA vasculitis (AAV).
  • CLEVELAND, Feb. 12, 2024 (GLOBE NEWSWIRE) --  NovelMed today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Ruxoprubart, an alternative pathway (AP) blocker anti-Bb antibody, for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
  • FDA's recognition of Ruxoprubart as an orphan drug for PNH underscores its potential to fulfill a crucial need for individuals grappling with this disease condition.

RTW Charitable Foundation Announces Six Research Grant Recipients

Retrieved on: 
Monday, February 12, 2024
Biotechnology, Health, Philanthropy, Pharmaceutical, Neurology, Research, Fund Raising, Genetics, Foundation, Science, Clinical Trials, Therapy, RTW, University College London, Grant (money), Metabolism, Toxicity, Doctor of Philosophy, IRF2BPL, Nationwide, University, Gene, Nonprofit organization, Growth, Hospital, Life, Weakness, Translation, University of California, Patient, NIHR, Child, AAV, Kidney transplantation, Kidney failure, Pharmaceutical industry

RTW Charitable Foundation (“RTWCF”) announced today that it has awarded $900,000 in research grants to support six research studies.

Key Points: 
  • RTW Charitable Foundation (“RTWCF”) announced today that it has awarded $900,000 in research grants to support six research studies.
  • “Optimizing AAV-SPL gene therapy for sphingosine phosphate lyase insufficiency syndrome” (Julie Saba, University of California, San Francisco, San Francisco, CA).
  • “RTW Charitable Foundation is proud to provide financial and scientific support to these six incredible researchers and their respective studies.
  • In addition to delivering financial resources, RTWCF also provides grant recipients with hands-on scientific support, helping them improve or further develop their research and research tools and identify new therapeutic targets.

Altamira Therapeutics’ Peptide-Based Delivery Platform Shown to Enhance Potency of Commonly Used Gene Delivery Method as Published in Peer-Reviewed Journal

Retrieved on: 
Wednesday, February 7, 2024
Vivisection, Peptide, Cell, RNA, Therapy, Alternative medicine, Recombinant, Safety, FDA, Dicarboxylic acid, AVV, Mouse, Food, AAV, Risk, Journal, Tissue, Cytoplasm, Melittin, Vaccine

HAMILTON, BERMUDA, Feb. 7, 2024 -- Altamira Therapeutics Ltd. (Nasdaq: CYTO) ("Altamira" or the "Company"), a company providing nanoparticle-based technology for efficient RNA delivery to extrahepatic targets, announced today the publication of a peer-reviewed article in the Journal of Integrative Medicine titled, "Melittin analog p5RHH enhances recombinant adeno-associated virus transduction efficiency". This work evaluates the use of various peptides to enhance adeno-associated virus (AAV) cell transduction and was conducted by an independent research group.1 Recombinant AAVs are commonly used as carriers to introduce nucleic acids in cells for gene therapy; several AAV-based gene therapy drugs have already been approved by the U.S. Food and Drug Administration (FDA).

Key Points: 
  • The study sought to find ways of increasing the endosomal release of AAV-based therapeutics by using peptides derived from melittin, a component of bee venom known for its ability to permeabilize biological membranes.
  • The research group evaluated 76 melittin derivatives, including p5RHH, the peptide underlying Altamira’s OligoPhore™ / SemaPhore™ nanoparticle platform for RNA delivery.
  • The scientists discovered that insertion of p5RHH into the AAV vector (p5RHH-rAAV) not only enhanced cell transduction, but also succeeded in transducing cell lines typically considered resistant to AAVs.
  • “Better transduction efficiency means that lower doses of AAVs may be used, which could lower the risk for deleterious immune responses and increase the safety of AAV-based vectors.

The Foundation for the National Institutes of Health Announces Publication of Regulatory Playbook to Advance AAV Gene Therapies for Rare Diseases

Retrieved on: 
Tuesday, February 6, 2024
Health, General Health, Partnership, AMP, Playbook, Knowledge, Investigational New Drug, Research, NIH, Government, Clinical trial, Bespoke Gene Therapy Consortium, Patient, IND, Patient safety, FDA, Publication, Investment, MPH, Food, AAV, Disease, Drug development, FNIH, Associate, Bangladesh Technical Education Board, Doctor of Philosophy, Organization, Pharmaceutical industry

The Foundation for the National Institutes of Health (FNIH) today announces the online publication of the first “playbook” designed to help accelerate the development of adeno-associated virus (AAV) gene therapies for rare diseases.

Key Points: 
  • The Foundation for the National Institutes of Health (FNIH) today announces the online publication of the first “playbook” designed to help accelerate the development of adeno-associated virus (AAV) gene therapies for rare diseases.
  • AMP BGTC is a public-private partnership between the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), life science companies, and patient foundations.
  • “Gene therapies offer promise to thousands of patients with rare diseases, but progress is slowed by uncertainties about manufacturing and a clear regulatory path.
  • The playbook was created as a “one-stop-shop guide” for clinical and drug development researchers who are working to bring AAV gene therapies to rare disease patients.

Ultragenyx Announces Data Demonstrating Treatment with UX111 Results in Significant Reduction in Heparan Sulfate Exposure in Cerebrospinal Fluid Correlated with Improved Long-term Cognitive Function in Patients with Sanfilippo Syndrome Type A (MPS IIIA)

Retrieved on: 
Tuesday, February 6, 2024
Sanfilippo, Family, Cognitive development, Research, Patient, Cerebrospinal fluid, HS, AAV, CSF, MPS, Mitt, Pharmaceutical industry

These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17).

Key Points: 
  • These data are from the modified intention to treat group (mITT) in the pivotal Transpher A study (N=17).
  • 8 of 17 patients in the mITT group who reached 24 months post-treatment achieved an overall mean percent reduction from baseline of 51% (p
  • Specifically, 15 of 17 patients had both a reduction in toxic CSF HS exposure and an improvement in cognitive function.
  • Both trials are ongoing, and patients will continue to be followed for a minimum of 5 years following treatment with UX111.

Viral Sensitizers Market Research Report 2023: Viral Vaccines, Oncolytic Viral Therapies and Viral Vectors - Global Industry Trends and Forecasts, 2020-2022 and 2023-2035 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, February 6, 2024
Health, Oncology, Other Health, Science Advisory Board, COVID-19, Genetic engineering, Workflow, Cell, Vector (mathematics and physics), Government, MLD, Cold medicine, Tetanus, Patient, DTP, Virus, COVID-19 vaccine, FDA, World Health Organization, AAV, Cell death, Vaccine, Infection, Measles, Gene, Viral, GMP, Immunization, WHO

The "Viral Sensitizers Market: Distribution by Application Area (Viral Vaccines, Oncolytic Viral Therapies and Viral Vectors) and Key Geographical Regions (North America, Europe and Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts, 2023-2035" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Viral Sensitizers Market: Distribution by Application Area (Viral Vaccines, Oncolytic Viral Therapies and Viral Vectors) and Key Geographical Regions (North America, Europe and Asia-Pacific and Rest of the World): Industry Trends and Global Forecasts, 2023-2035" report has been added to ResearchAndMarkets.com's offering.
  • The market research report highlights the efforts of several stakeholders engaged in this rapidly evolving segment of the viral manufacturing industry.
  • Key takeaways of the viral sensitizers market report are briefly discussed below.
  • We have extensively studied the historical market data within this industry, in order to develop a deeper understanding of the evolutionary market trends.

Taysha Gene Therapies Announces Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Friday, February 2, 2024
Employment, CNS, AAV, Compensated emancipation, Central nervous system

DALLAS, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that, on February 1, 2024, the Compensation Committee of Taysha's Board of Directors granted two new employees, in the aggregate, options to purchase 180,000 shares of the Company's common stock in connection with their employment.

Key Points: 
  • DALLAS, Feb. 02, 2024 (GLOBE NEWSWIRE) -- Taysha Gene Therapies, Inc. (Nasdaq: TSHA), a clinical-stage gene therapy company focused on developing and commercializing AAV-based gene therapies for the treatment of monogenic diseases of the central nervous system (CNS), today announced that, on February 1, 2024, the Compensation Committee of Taysha's Board of Directors granted two new employees, in the aggregate, options to purchase 180,000 shares of the Company's common stock in connection with their employment.
  • The stock options were granted under the Taysha Gene Therapies, Inc. 2023 Inducement Plan as an inducement material to the individuals entering employment with Taysha in accordance with Nasdaq Listing Rule 5635(c)(4).
  • The stock options have an exercise price of $1.56 per share, which is equal to the closing price of Taysha's common stock on the date of grant.
  • Vesting of the stock option is subject to such employee's continued service to Taysha on each vesting date.

Muscular Dystrophy Association Announces Recipient of 2024 MDA Legacy Award for Achievement in Research, is Jeffrey Chamberlain, Ph.D., Leading Professor in Gene Therapy

Retrieved on: 
Tuesday, January 30, 2024
Vivisection, Muscular dystrophy, Hospital, Neuromuscular disease, Immune system, Episcopal conference, MDA, MD, Muscular Dystrophy Association, Patient, Reward, AAV, Protein, Regenerative medicine, DMD, Duchenne muscular dystrophy, Doctor of Philosophy, University, Conference, Vaccine

The award will be presented at the 2024 MDA Clinical & Scientific Conference on Monday, March 4, by Louis Kunkel, PhD , of Boston Children’s Hospital.

Key Points: 
  • The award will be presented at the 2024 MDA Clinical & Scientific Conference on Monday, March 4, by Louis Kunkel, PhD , of Boston Children’s Hospital.
  • The MDA Legacy Award for Achievement in Research is an annual recognition for outstanding accomplishments in neuromuscular disease research.
  • "The Muscular Dystrophy Association is elated to celebrate Dr. Chamberlain and honor him with the 2024 MDA Legacy Award for Achievement in Research for his achievements in translational research.
  • The previous recipient of the MDA Legacy Award for Achievement in Research in 2023 was Merit Cudkowicz, MD, MSC Massachusetts General Hospital, for her achievements in clinical research.

Propelling Diagnostics and Life Sciences Innovation from Early Phase Research to the Clinic: Revvity Showcases Cutting-Edge Solutions at SLAS2024

Retrieved on: 
Friday, February 2, 2024
Science, Radiology, Biotechnology, Research, Pharmaceutical, Health, Medical Devices, Health Technology, Transverse mode, Drug discovery, RNA interference, AAV, HCS, Drug development, PLX, Medicine, Genomics, CDX, CRISPR, G3, NGS, SLAS, GMP, Automation, Flow cytometry, Omics, Engineering, TB

As the landscape of medicine becomes more personalized, Revvity aims to provide high-value, cutting-edge solutions that foster scientific innovation and positively impact human health.

Key Points: 
  • As the landscape of medicine becomes more personalized, Revvity aims to provide high-value, cutting-edge solutions that foster scientific innovation and positively impact human health.
  • Its offerings are designed to provide differentiated capabilities, providing customers with tools needed for success in precision medicine and beyond.
  • Dr. Alan Fletcher, Revvity’s senior vice president, life sciences, stated, "We're here in our backyard showing how we're seamlessly bringing it all together, powering innovation from research to reality.
  • Revvity will be exhibiting a selection of its extensive range of life sciences and diagnostics products and services.