NHP

Vaxart Provides Business Update and Reports Full Year 2023 Financial Results

Retrieved on: 
Thursday, March 14, 2024
Mother, Research, Breast milk, Conference call, Development, Bill, Webcast, COVID-19, SAN, VOC, GI, Messenger, XBB, NHP, SARS-CoV-2, Insurance, Investment, Clinical trial, Antibody, Safety, Vaxart, Nextgen, Vaccine, FDA, Food, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., March 14, 2024 (GLOBE NEWSWIRE) -- Vaxart, Inc. (Nasdaq: VXRT) today announced its business update and financial results for the full year 2023.

Key Points: 
  • ET
    SOUTH SAN FRANCISCO, Calif., March 14, 2024 (GLOBE NEWSWIRE) -- Vaxart, Inc. (Nasdaq: VXRT) today announced its business update and financial results for the full year 2023.
  • Financial Results for the Full Year Ended December 31, 2023
    Cash, cash equivalents and investments totaled $39.7 million as of December 31, 2023.
  • Vaxart reported a net loss of $82.5 million for the full year 2023, compared to $107.8 million for the full year 2022.
  • The Vaxart senior management team will host a conference call to discuss the business update and financial results for the full year 2023 today, beginning at 4:30 p.m.

Precision BioSciences Reports Fourth Quarter and Fiscal Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 27, 2024
Technology, Biotechnology, FDA, Nanotechnology, Health, Pharmaceutical, Stem Cells, Other Science, Research, Infectious Diseases, Genetics, Fitness & Nutrition, Science, Clinical Trials, Janus, Vivo, Gene editing, Precision BioSciences, Patient, Cash, Mitochondrion, Health care, Infant, NHP, IND, Duchenne muscular dystrophy, Investment, Therapy, ATM, Lilly, Precision, Cell, Skeletal muscle, Hepatitis B, Rachel Haurwitz, Central nervous system, Novartis, Primate, RNA, DNA, Mitochondrial disease, Hepatology, CRISPR, Safety, VWAP, PMM, Food, AAV, Development, Prevail, Publishing, Gene, ASX, University, Diaphragm, OTC, Hemoglobinopathy, IMU, Economics, Administration, Sickle cell disease, PCSK9, CTA, MHRA, Reindeer, Heart, Autoimmune disease, Eli Lilly and Company, DMD

PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B.

Key Points: 
  • PBGENE-HBV (Viral Elimination Program): Precision is developing PBGENE-HBV for the treatment of patients with chronic hepatitis B.
  • In November 2023, Precision presented preclinical efficacy and safety data at the 2023 American Association for the Study of Liver Diseases Annual Meeting.
  • Cash and Cash Equivalents: As of December 31, 2023, Precision had $116.7 million in cash and cash equivalents.
  • Revenues: Total revenues for the quarter ended December 31, 2023 were $7.0 million, as compared to $10.6 million for the quarter ended December 31, 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.

Empowering Women in the Natural Health Product Industry on International Women’s Day

Retrieved on: 
Friday, March 8, 2024
International, CHFA, Woman, Health Canada, Synovial joint, Regulatory affairs, NHP, Health, Policy, Internet, Dietary supplement

Health Canada’s proposed regulations will decimate the NHP industry, disproportionately impacting women and women-led businesses.

Key Points: 
  • Health Canada’s proposed regulations will decimate the NHP industry, disproportionately impacting women and women-led businesses.
  • This International Women’s Day, the Canadian Health Food Association (CHFA) is calling on the federal government to press pause.
  • “Many of these businesses will be forced to close.”
    It's a well-known fact that women have been historically neglected in health research, and Health Canada’s latest regulatory reform on NHPs is no exception.
  • Women are much more likely to purchase NHPs, but Health Canada’s changes will take safe, compliant Canadian products off Canadian shelves.

Paratek Pharmaceuticals Announces Positive Efficacy Data for NUZYRA® as Post-Exposure Prophylaxis of Inhalational Anthrax, Triggering Additional Procurement under BARDA Project BioShield Contract

Retrieved on: 
Tuesday, March 5, 2024
Human services, Anthrax, Soil, FDA, Shanda, NHP, PEP, Survival rate, Administration, Post-exposure prophylaxis, Observation, Death, Bioterrorism, Infection, Tablet, Development, Partnership, Pharmaceutical industry

Anthrax (B. anthracis) is an infectious disease caused by gram-positive, rod-shaped, spore-forming bacteria that can lead to severe illness and death.

Key Points: 
  • Anthrax (B. anthracis) is an infectious disease caused by gram-positive, rod-shaped, spore-forming bacteria that can lead to severe illness and death.
  • Oral NUZYRA tablets associated with this procurement were manufactured in the United States as part of the company’s U.S. onshoring efforts.
  • “This procurement also represents a significant milestone in the company’s onshoring efforts as for the first time Paratek is providing BARDA with NUZYRA tablets produced on U.S. soil.
  • In December 2019, BARDA awarded Paratek a contract (75A50120C00001) that is now valued at up to approximately $304 million.

Sana Biotechnology Reports Fourth Quarter and Full Year 2023 Financial Results and Business Updates

Retrieved on: 
Thursday, February 29, 2024
Neoplasm, CD47, VIVID, CD19, Exercise, Science Translational Medicine, Bone marrow, Lymphoid leukemia, Lupus nephritis, CTA, Biotechnology, Escape Room, Research and development, GLEAM, ANCA, GAAP, Sana, Insulin, Development, Immune system, Regeneration, ASH, CD22, AACR, B cell, Safety, Immunosuppression, Diabetes, Patent, IND, Insurance, Nature Biotechnology, NHL, Cancer, CLL, Contingency, Cell, NHP, Organoid, Autoimmunity, Administration, Cell Stem Cell, Patient, HIP, Nature Communications, ISSCR, C-peptide, Vaccine

The Investigational New Drug Application (IND) cleared in 4Q 2023, and Sana expects to share initial data in 2024.

Key Points: 
  • The Investigational New Drug Application (IND) cleared in 4Q 2023, and Sana expects to share initial data in 2024.
  • The Clinical Trial Application (CTA) cleared in 4Q 2023, and Sana expects initial data to be shared in the first half of 2024.
  • Non-GAAP research and development expense in 2023 excludes an expense related to the impairment of certain lab equipment and leasehold improvements, primarily due to the portfolio prioritization in the fourth quarter of 2023.
  • A discussion of non-GAAP measures, including a reconciliation of GAAP and non-GAAP measures, is presented below under “Non-GAAP Financial Measures.”

Sana Biotechnology Announces Publication of Preclinical Diabetes Data in Cell Stem Cell Demonstrating Insulin Independence Following Transplantation of Hypoimmune Allogeneic Primary Islet Cells Without Immunosuppression in a Diabetic NHP

Retrieved on: 
Tuesday, February 13, 2024
JDRF, Diabetes, Biotechnology, Regeneration, Doctor of Philosophy, IST, MD, Escape Room, Research, Behavior informatics, CD47, Cell, NHP, Organoid, Safety, Transplant, Immunosuppression, Cell Stem Cell, Patient, Insulin, HIP, T1D, Glucose, Pharmaceutical industry

SEATTLE, Feb. 13, 2024 (GLOBE NEWSWIRE) -- Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, today announced that Cell Stem Cell has published a paper titled “Hypoimmune islets achieve insulin independence after allogeneic transplantation in a fully immunocompetent non-human primate.” The paper evaluated a transplant of Sana’s engineered allogeneic, hypoimmune (HIP)-modified pancreatic islet cells into a fully immunocompetent, diabetic non-human primate (NHP). These modified islet cells, which cluster into effective endocrine organoids, are termed “pseudo islet grafts” (p-islets). The results demonstrated that the HIP-modified p-islets engrafted following intramuscular injection and provided stable endocrine function, enabling insulin independence in the absence of immunosuppression.

Key Points: 
  • These modified islet cells, which cluster into effective endocrine organoids, are termed “pseudo islet grafts” (p-islets).
  • The results demonstrated that the HIP-modified p-islets engrafted following intramuscular injection and provided stable endocrine function, enabling insulin independence in the absence of immunosuppression.
  • This publication, along with the ongoing IST, will provide invaluable insights toward our stem cell derived product candidate, SC451.
  • It was thus demonstrated that the tightly controlled blood glucose levels and insulin independence was entirely due to well-functioning HIP p-islets.

Jaguar Gene Therapy Announces FDA Clearance of IND to Study JAG201 in a Genetic Form of Autism Spectrum Disorder and Phelan-McDermid Syndrome

Retrieved on: 
Wednesday, January 31, 2024
FDA, Other Health, Pharmaceutical, Managed Care, General Health, Biometrics, Mental Health, Infectious Diseases, Neurology, Genetics, Clinical Trials, Biotechnology, Science, Other Science, Research, Health, Parent, Synapse, Neuroscience, NHP, Mutation, Spectrum, Genetic disorder, Quality of life, Learning, PMSF, Memory, ASD, PMS, ICV, Investigational New Drug, Cognitive science, Broad, Prevalence, IND, Jaguar, Genetic testing, Frasier, SHANK3, ID, Spinal cord, Haploinsufficiency, DSM-IV codes, Gene therapy, Patient, Central nervous system, Harvard University, Speech, Food, Neuron, Disease, Communication, Risk, Sibling, Disorder, Psychiatry, 22q13 deletion syndrome, Intellectual disability, Broad Institute, MIT, Pediatrics, Pharmaceutical industry

Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).

Key Points: 
  • Jaguar Gene Therapy, a biotechnology company accelerating breakthroughs in gene therapy for patients suffering from severe genetic diseases, including those that affect sizeable patient populations, today announced the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) Application for JAG201, a gene therapy for a genetic form of autism spectrum disorder (ASD) and Phelan-McDermid syndrome (PMS).
  • JAG201 aims to deliver functional SHANK3 via the AAV9 vector to treat the root cause of the disease.
  • “We are pleased to receive FDA clearance to bring our investigational SHANK3 gene therapy to the clinic.
  • The pre-clinical data indicate that JAG201 may have the potential to be transformative for those suffering with the disorder,” said Joe Nolan, chief executive officer of Jaguar Gene Therapy.

ProQR Highlights New Platform Data from Presentation on Axiomer™ RNA Editing Technology at Deaminet 2024

Retrieved on: 
Friday, January 19, 2024
EON, RNA-Seq, Oligonucleotide, Knowledge, RNA, Science, Circulatory system, Mouse, NHP, NTCP, ACTB, Cholestasis, ANGPTL3, Cardiovascular disease, Plasma, Conference, B4GALT1, LDL, Liver, Eon, ProQR, ADAR, Nested polymerase chain reaction, CNS, Video game

“We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.

Key Points: 
  • “We were pleased to present new in vivo data for our proprietary Axiomer RNA editing technology platform at the Deaminet 2024 meeting, demonstrating robust editing of ACTB in the liver of NHP, as well as functional protein data with the liver target ANGPTL3 in mice,” said Gerard Platenburg, Chief Scientific Officer of ProQR.
  • “To date, we have generated robust in vitro and in vivo preclinical platform data across a range of liver and CNS targets.
  • Presentation of new data highlighting up to 70% editing efficiency of ACTB in liver of mice and NHPs at multiple timepoints.
  • These new preclinical platform data further highlight the potential of Axiomer EONs in preparation for clinical development and supporting discovery of potential new therapeutic applications.

Wave Life Sciences Highlights Recent Achievements and Upcoming 2024 Milestones

Retrieved on: 
Monday, January 8, 2024
Well, RNA, Conference, Science, Wave, Messenger, Research, AAT, Chemistry, Pizzicato, Small RNA, Lung, Weight, Health, Disease, MBA, MD, Mouse, HD, Human, Serum, GSK, Mutation, Genetics, Coronary artery disease, Obesity, Genomics, Concentration ratio, Central nervous system, Patient, Liver disease, Lof, HTT, Medicine, Therapy, Oligonucleotide, NHP, CTA, Vaccine

We expect 2024 will be an inflection year that will drive significant value for Wave, our shareholders, and most importantly, for the patients who will benefit from our research,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.

Key Points: 
  • We expect 2024 will be an inflection year that will drive significant value for Wave, our shareholders, and most importantly, for the patients who will benefit from our research,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences.
  • Expected milestones: Wave plans to deliver proof-of-mechanism data in individuals with AATD in 2024.
  • Expected milestones: Wave expects to deliver data, including dystrophin protein expression from muscle biopsies at 24 weeks, in the third quarter of 2024.
  • A live webcast of this presentation will be available on the Investor Relations page of the Wave Life Sciences website at http://ir.wavelifesciences.com .