Investigational New Drug

Genflow Biosciences Releases 2023 Year-End Review and Highlights Key Priorities for 2024

Retrieved on: 
Wednesday, February 7, 2024
SIRT6, Fatty liver disease, Investigational New Drug, Research, Therapy, CDMO, Patient, IND, Longevity, EMA, Patent Cooperation Treaty, QMS, Medication, European Medicines Agency, Werner syndrome, NASH, GMP, PCT, Pharmaceutical industry

LONDON, Feb. 07, 2024 (GLOBE NEWSWIRE) -- Genflow Biosciences Plc (Genflow or the "Company”) (LSE:GENF) (OTCQB:GENFF), an emerging leader in the field of longevity research developing therapeutics that potentially halt or slow the ageing process is pleased to provide a year-end review and summary of its progress during 2023 and defined strategic priorities for 2024.

Key Points: 
  • LONDON, Feb. 07, 2024 (GLOBE NEWSWIRE) -- Genflow Biosciences Plc (Genflow or the "Company”) (LSE:GENF) (OTCQB:GENFF), an emerging leader in the field of longevity research developing therapeutics that potentially halt or slow the ageing process is pleased to provide a year-end review and summary of its progress during 2023 and defined strategic priorities for 2024.
  • Genflow expanded its intellectual property portfolio with a provisional patent application for editing the SIRT6 gene, linked to longevity and age-related diseases.
  • Began trading on the OTCQB Venture Market in the US under the ticker GENFF, aligned to its US-focused strategy.
  • Dr. Eric Leire, CEO of Genflow commented: “The milestones we reached in 2023 were numerous, and I commend our team for their exceptional commitment to execution and delivery.

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.

Breaking Ground in Prostate Cancer: BriaCell Announces Lead Prostate Cancer Candidate Bria-Pros+, Initiates GMP Manufacturing

Retrieved on: 
Tuesday, February 6, 2024
Prostate cancer, Investigational New Drug, Cytokine, Immunotherapy, SITC, Clinical trial, NK, Society, Patient, IND, Good, Death, TSX, Allele, NKT, Therapy, Vaccine, Good manufacturing practice, HLA, Cancer, GMP, BCT

We are enthusiastic about commencing GMP manufacturing for our prostate cancer clinical candidate, continuing our efforts at transforming targeted cancer therapy via the introduction of potent personalized cellular cancer vaccines,” stated Dr. William V. Williams, BriaCell’s President and CEO.

Key Points: 
  • We are enthusiastic about commencing GMP manufacturing for our prostate cancer clinical candidate, continuing our efforts at transforming targeted cancer therapy via the introduction of potent personalized cellular cancer vaccines,” stated Dr. William V. Williams, BriaCell’s President and CEO.
  • “The successful development of this first candidate showcases our capacity to create novel cancer immunotherapies and advance them towards clinical application.
  • BriaCell genetically engineers cancer cell lines to produce cytokines and co-stimulatory factors that significantly increase immune stimulation compared to the unmodified (parent) cancer cell lines.
  • According to 2024 Cancer Facts & Figures , prostate cancer is projected to be the most common cancer among men in 2024.

Veru Announces FDA Clearance of IND Application to Initiate Phase 2b Clinical Trial with Enobosarm to Treat Muscle Loss Associated with Weight Loss Drugs

Retrieved on: 
Tuesday, February 6, 2024
Weight, Weight loss, Atrophy, Calendar, Investigational New Drug, Overweight, Trial of the century, Patient, IND, Safety, Facial muscles, FDA, Food, Muscle weakness, Frailty, Metabolism, SARM, Dietary supplement

MIAMI, FL, Feb. 06, 2024 (GLOBE NEWSWIRE) -- Veru Inc. (NASDAQ: VERU), a late clinical stage biopharmaceutical company focused on developing innovative medicines for potentially higher quality weight loss, oncology, and viral induced acute respiratory distress syndrome (ARDS), today announced that the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application for its Phase 2b clinical study to evaluate enobosarm, an oral novel selective androgen receptor modulator (SARM), to preserve muscle mass and physical function and further increase fat loss in patients receiving a Glucagon-like peptide-1 receptor agonist (GLP-1 RA) drug for potentially higher quality weight loss.

Key Points: 
  • GLP-1 RA drugs are very effective drugs that result in significant weight loss.
  • Unfortunately, studies have shown that up to 50% of the total weight loss comes from muscle which is problematic as muscle is essential for metabolism, strength, and physical function.
  • The primary endpoint is lean body mass (muscle), and the key secondary endpoint is total body fat mass at 16 weeks.
  • The results of the separate Phase 2b open label extension clinical study is expected in calendar Q2 2025.

Aprea Therapeutics Announces Submission of IND Application for APR-1051, a Next Generation WEE1 Kinase Inhibitor

Retrieved on: 
Tuesday, February 6, 2024
APRE, Investigational New Drug, DNA, Patient, IND, Safety, FDA, PLK, Therapy, Non-Euclidean geometry, WEE1, Pharmaceutical industry

APR-1051 is an oral inhibitor of WEE1 kinase, which plays important role in cell cycle regulation and DNA damage repair.

Key Points: 
  • APR-1051 is an oral inhibitor of WEE1 kinase, which plays important role in cell cycle regulation and DNA damage repair.
  • * Aprea has conducted extensive pre-clinical studies with APR-1051, which have demonstrated that the molecule may have highly potent anti-tumor activity, with a potentially favorable pharmacokinetic (PK) profile.
  • “Submission of an IND represents an important milestone for our APR-1051 development program,” said Dr. Oren Gilad, President and CEO of Aprea.
  • “APR-1051 is a next generation inhibitor of WEE1 kinase and, based on its unique characteristics, we believe it will be best in class.

Scholar Rock Presents New Preclinical Data Demonstrating Potential Benefit of SRK-439 for Healthy Weight Loss Management

Retrieved on: 
Tuesday, February 6, 2024
Biotechnology, Health, Fitness & Nutrition, Pharmaceutical, Clinical Trials, Poster, Weight loss, Pharmacokinetics, Human, Investigational New Drug, Obesity, Patient, IND, Fasting, Keystone, Myostatin, GDF11, Mouse, Rock, MPH, Food, SMA, Body composition, Immunoglobulin G, Nandi (Hinduism), Doctor of Philosophy, Liraglutide, ELISA, Dietary supplement

“These preclinical data showing that SRK-439 preserves lean mass and improves fat mass loss provide compelling scientific rationale to study SRK-439 in combination with GLP-1 RA therapies for healthy weight loss management,” said Jay Backstrom, M.D., MPH, President and CEO of Scholar Rock.

Key Points: 
  • “These preclinical data showing that SRK-439 preserves lean mass and improves fat mass loss provide compelling scientific rationale to study SRK-439 in combination with GLP-1 RA therapies for healthy weight loss management,” said Jay Backstrom, M.D., MPH, President and CEO of Scholar Rock.
  • “We believe that preserving lean muscle mass through our highly selective approach to myostatin inhibition in combination with GLP-1 RA therapy has the potential to transform the management of weight loss.
  • In parallel, Scholar Rock is developing SRK-439, a novel investigational selective myostatin inhibitor, optimized for the treatment of obesity.
  • SRK-439 also improved fat mass loss (–36.60% to –46.32% from baseline with semaglutide; –17.31% to –19.04% from baseline with liraglutide).

Deciphera Pharmaceuticals Announces Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Tuesday, February 6, 2024
Biotechnology, Pharmaceutical, Health, Oncology, OS, KIT, European, Investigational New Drug, Trial of the century, DNA, Research, Patient, IND, Autophagy, ASCO, GIST, Public expenditure, FDA, EMA, MAA, Part, Food, Society, TGCT, Gastrointestinal stromal tumor, European Medicines Agency, Sunitinib, Neoplasm, Cancer, INSIGHT, Tenosynovial giant cell tumor, NDA, Nature Medicine, Pharmaceutical industry

Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, today announced financial results for the fourth quarter and year ended December 31, 2023 and provided a corporate update.

Key Points: 
  • Deciphera Pharmaceuticals, Inc. (NASDAQ: DCPH), a biopharmaceutical company focused on discovering, developing, and commercializing important new medicines to improve the lives of people with cancer, today announced financial results for the fourth quarter and year ended December 31, 2023 and provided a corporate update.
  • Expects to present updated results from the Phase 1/2 study of vimseltinib in TGCT in the second half of 2024.
  • Expects to initiate a Phase 2 proof-of-concept study of vimseltinib for the treatment of chronic graft versus host disease (cGVHD) in the fourth quarter of 2024.
  • Cost of Sales: Cost of sales were $1.8 million in the fourth quarter of 2023, which includes $0.9 million in cost of product sales, compared to cost of product sales of $0.7 million for the fourth quarter of 2022.

Genflow Biosciences Releases 2023 Year-End Review and Highlights Key Priorities for 2024

Retrieved on: 
Thursday, February 1, 2024
SIRT6, Fatty liver disease, Investigational New Drug, Research, Therapy, CDMO, Patient, IND, Longevity, EMA, Patent Cooperation Treaty, QMS, Medication, European Medicines Agency, Werner syndrome, NASH, GMP, PCT, Pharmaceutical industry

LONDON, Feb. 01, 2024 (GLOBE NEWSWIRE) -- The Company (LSE:GENF) (OTCQB:GENFF), an emerging leader in the field of longevity research developing therapeutics that potentially halt or slow the ageing process, is pleased to provide a year-end review and summary of its progress during 2023 and defined strategic priorities for 2024.

Key Points: 
  • The full year-end review can be viewed on Genflow’s website: https://genflowbio.com/genflow-biosciences-plc-2023-year-in-review/.
  • Genflow expanded its intellectual property portfolio with a provisional patent application for editing the SIRT6 gene, linked to longevity and age-related diseases.
  • Began trading on the OTCQB Venture Market in the US under the ticker GENFF, aligned to its US-focused strategy.
  • Dr. Eric Leire, CEO of Genflow commented: “The milestones we reached in 2023 were numerous, and I commend our team for their exceptional commitment to execution and delivery.

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.

2024 OREF Clinical Research Award Celebrates Study of Local Gene Therapy to Treat Osteoarthritis

Retrieved on: 
Friday, February 2, 2024
University, Visual analogue scale, DSM-IV codes, Cell, Investigational New Drug, Mayo Clinic, Research, Liver, Knee, DNA, Disease, OA, Patient, Pain, Safety, University of Minnesota, Swelling, FDA, Rheumatoid arthritis, Osteoarthritis, Arthritis, Food, Risk, DRS, Biology, WOMAC, University of Florida, Doctor of Philosophy, Cancer, Joint, Molecular medicine, Pharmaceutical industry

ROSEMONT, Ill. , Feb. 2, 2024 /PRNewswire/ -- The 2024 Orthopaedic Research and Education Foundation (OREF) Clinical Research Award was presented to Christopher H. Evans, PhD (Mayo Clinic), Steven C. Ghivizzani, PhD (University of Florida), and Paul D. Robbins, PhD (University of Minnesota), for their research on local gene therapy for osteoarthritis (OA).

Key Points: 
  • ROSEMONT, Ill. , Feb. 2, 2024 /PRNewswire/ -- The 2024 Orthopaedic Research and Education Foundation (OREF) Clinical Research Award was presented to Christopher H. Evans, PhD (Mayo Clinic), Steven C. Ghivizzani, PhD (University of Florida), and Paul D. Robbins, PhD (University of Minnesota), for their research on local gene therapy for osteoarthritis (OA).
  • Together, the team spearheaded the research from a laboratory concept to human clinical trials over the course of 30 years.
  • The OREF Award recognizes outstanding clinical research related to musculoskeletal disease or injury.
  • Evans, Ghivizzani and Robbins co-founded an arthritis gene therapy startup company, Genascence Corporation, which will help fund future clinical trials.