Disorder

Neurocrine Biosciences Presents CAHtalyst™ Adult Study Baseline Characteristics and Data on Impact of Supraphysiologic Glucocorticoid Therapy at AACE 2024

Retrieved on: 
Donnerstag, Mai 9, 2024
Disorder, Common, Risk, GC, Overweight, Trial of the century, Adrenocorticotropic hormone, Hyperlipidemia, Associate, Deficit spending, Hydrocortisone, Neurocrine Biosciences, White matter, Food, Oklahoma State University–Stillwater, Cognition, Psychology, Testosterone, Male, Literature, Brain, DSM-IV codes, A4, SAN, GCS, CAH, Acne, Safety, Glucocorticoid, Research, Depression, Congenital adrenal hyperplasia, Osteopenia, Anxiety, Division, Hirsutism, Androstenedione, Psychiatry, Psychosis, Hypertension, Metabolism, Poster, Pharmaceutical industry

The CAHtalyst Adult study baseline data demonstrate the potential long-term consequences of current CAH treatments, with many patients in young adulthood experiencing disorders found more commonly in people decades older, including osteopenia, hypertension and hyperlipidemia.

Key Points: 
  • The CAHtalyst Adult study baseline data demonstrate the potential long-term consequences of current CAH treatments, with many patients in young adulthood experiencing disorders found more commonly in people decades older, including osteopenia, hypertension and hyperlipidemia.
  • These new data were presented at oral presentations and poster sessions at the American Association of Clinical Endocrinology 2024 Annual Meeting in New Orleans.
  • Despite supraphysiologic GC dosing, levels of adrenocorticotropic hormone, 17-hydroxyprogesterone and androstenedione (A4) were elevated at baseline, with levels of testosterone (females) and A4/testosterone (males) also elevated.
  • The data from both studies supported two New Drug Applications submitted to the U.S. Food and Drug Administration in April 2024.

Epitor Therapeutics Unveils CasNano: A Compact, All-in-One CRISPR/Cas for Single AAV Therapies at the American Society of Gene & Cell Therapy Annual Meeting

Retrieved on: 
Donnerstag, Mai 9, 2024
Dicarboxylic acid, Disorder, CRISPR, Cas, RNA, Cell, AAV, DNA, TET, Demethylation, Gene, Society, Annual general meeting, Therapy, Biotechnology

NEW YORK, May 9, 2024 /PRNewswire/ -- Epitor Therapeutics, a biotechnology pioneer in epigenetic editing, announces the creation of CasNano, a groundbreaking ultracompact Cas protein.

Key Points: 
  • NEW YORK, May 9, 2024 /PRNewswire/ -- Epitor Therapeutics, a biotechnology pioneer in epigenetic editing, announces the creation of CasNano, a groundbreaking ultracompact Cas protein.
  • The company unveiled new data during the 27th American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, showcasing over a 20-fold improvement in targeting efficiency via directed evolution of CasNano and innovative re-engineering of the tracrRNA component of the guide RNA.
  • "In addition to its nuclease activity, CasNano can efficiently demethylate, methylate, and activate target genes when catalytically deactivated, with protein effectors attached.
  • "CasNano is 70% smaller than spCas9 and has been engineered to optimize DNA targeting," added Epitor CEO, Dr. Megan McGill.

Keros Therapeutics Reports Recent Business Highlights and First Quarter 2024 Financial Results

Retrieved on: 
Mittwoch, Mai 8, 2024
Disorder, Protein, ATS, Investment, Pulmonary hypertension, Food, Research, Circulatory system, Conference, Orthostatic hypertension, KROS, Syndrome, Therapy, Patient, American Thoracic Society, Pharmaceutical industry

“Keros remained focused on clinical execution and continued to make strong progress across our pipeline in the first quarter of 2024,” said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer.

Key Points: 
  • “Keros remained focused on clinical execution and continued to make strong progress across our pipeline in the first quarter of 2024,” said Jasbir S. Seehra, Ph.D., President and Chief Executive Officer.
  • General and administrative expenses were $10.3 million for the first quarter of 2024 as compared to $7.8 million for the same period in 2023.
  • Keros’ cash and cash equivalents as of March 31, 2024 was $442.4 million compared to $331.1 million as of December 31, 2023.
  • Keros expects that the cash and cash equivalents it had on hand at March 31, 2024 will enable Keros to fund its operating expenses and capital expenditure requirements into 2027.

NMDP’s Amy Ronneberg Recognized as Most Admired CEO by Minneapolis/St. Paul Business Journal

Retrieved on: 
Montag, Mai 6, 2024
Breast cancer, Volunteering, CFO, Disorder, Congress, Research, History, Female, Minneapolis–Saint Paul, Patient, Bone marrow, Committee on Finance, Journal, Management

MINNEAPOLIS, May 06, 2024 (GLOBE NEWSWIRE) -- NMDPSM, a global nonprofit leader in cell therapy, announced today that the Minneapolis / St. Paul Business Journal named CEO Amy Ronneberg to its 2024 list of Most Admired CEOs, an honor designated annually to 10 chief executives in the Twin Cities.

Key Points: 
  • MINNEAPOLIS, May 06, 2024 (GLOBE NEWSWIRE) -- NMDPSM, a global nonprofit leader in cell therapy, announced today that the Minneapolis / St. Paul Business Journal named CEO Amy Ronneberg to its 2024 list of Most Admired CEOs, an honor designated annually to 10 chief executives in the Twin Cities.
  • The Journal will celebrate these leaders’ organizational and business acumen as well as community and industry achievements at a ceremony in late September.
  • “I’m humbled and honored to be recognized among this stellar class of 2024 honorees,” Ronneberg said.
  • Ronneberg embodies NMDP’s vision to create a world where every patient can receive their life-saving cell therapy, impacting over 26,000 lives since becoming CEO.

PLK Targeted Therapies Market & Clinical Trials 2024: Onvansertib Leading the Way in PLK1 Inhibition, Pioneering Breakthroughs in Small Cell Lung Cancer and Chronic Myelomonocytic Leukemia Treatment - ResearchAndMarkets.com

Retrieved on: 
Dienstag, Mai 7, 2024
Health, Clinical Trials, Cytokinesis, Immunotherapy, Cancer, Chronic myelomonocytic leukemia, Inflammation, Disorder, Safety, Breast cancer, Viral, Apoptosis, SCLC, Cell proliferation, Research, Cell cycle, Carcinoma, DNA, Mitosis, Indication, Patient, Rheumatoid arthritis, Hepatitis B virus, Virus, Proteolysis targeting chimera, Multiple sclerosis, HIV, Messenger, PLK, Lupus, Genome, Biomarker, Medicine, PLK1, DA, HBV, Pharmaceutical industry

The "Global PLK Targeted Therapies Market Opportunity & Clinical Trials Insight 2024" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global PLK Targeted Therapies Market Opportunity & Clinical Trials Insight 2024" report has been added to ResearchAndMarkets.com's offering.
  • Onvansertib, a PLK1 inhibitor developed by Cardiff Oncology, is currently the most advanced candidate in clinical trials.
  • The progression of these candidates through the pipeline reflects the growing interest and confidence in PLK targeted therapies.
  • In conclusion, PLK targeted therapies are a rapidly developing field with revolutionary promise across a wide range of disease areas.

Rocket Pharmaceuticals Reports First Quarter 2024 Financial Results and Highlights Recent Progress

Retrieved on: 
Montag, Mai 6, 2024
Research, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, Education, FDA, CFO, Disorder, Regenerative medicine, University of California, Alexion Pharmaceuticals, PDUFA, Arena Pharmaceuticals, Danon disease, European Medicines Agency, Strategic planning, UCLA, Food, Molecule, Stanford University, Empire, BLA, Medication, Corporate communication, Definitive, Fanconi anemia, Patient, Increment, Safety, Rare disease, Regeneron Pharmaceuticals, AAV, Immunology, EMA, FA, Division, PKD, University, Marketing, MAA, Cell, Biologics license application, Pharmacology, Rare Disease Day, Disease, Mirati Therapeutics, Haematopoietic system, Gene, Society, Pyruvate kinase deficiency, Liberty Science Center, Annual general meeting, Pharmaceutical industry

Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending March 31, 2024.

Key Points: 
  • Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, late-stage biotechnology company advancing a sustainable pipeline of genetic therapies for rare disorders with high unmet need, today reported financial and recent operational results for the quarter ending March 31, 2024.
  • In April, Rocket announced that the European Medicines Agency (EMA) accepted the Marketing Authorization Application (MAA) for RP-L102 for the treatment of FA.
  • MAA acceptance was based on positive, previously disclosed data from the global RP-L102 Phase 1/2 clinical trial.
  • Mr. Ondrey was previously the CFO at Mirati Therapeutics and has held multiple senior finance leadership positions at Arena Pharmaceuticals, Alexion Pharmaceuticals, and Regeneron Pharmaceuticals.

Tyra Biosciences Announces Appointments of Susan Moran, M.D., M.S.C.E. and S. Michael Rothenberg, M.D., Ph.D. to its Board of Directors

Retrieved on: 
Dienstag, Mai 7, 2024
Stanford University, University, FGFR, Disorder, Fibroblast growth factor receptor, Hospital, NDA, QED, Achondroplasia, Hematology, Bile duct, Drug, Research and development, MAA, Yale University, RET, Growth, Duke University, Biotechnology, Therapy, Faculty, Osteochondrodysplasia, Harvard Medical School, Multiple sclerosis, Biology, Drug development, Drug discovery, Liver cancer, Bristol Myers Squibb, Massachusetts General Hospital, Puma Biotechnology, Pharmaceutical industry

and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.

Key Points: 
  • and S. Michael Rothenberg, M.D., Ph.D. as independent directors, and the resignation of Isan Chen, M.D.
  • "Susan and Michael's impressive careers will provide TYRA with valuable guidance as we advance our potential next-generation precision candidates toward people in need.
  • In connection with these appointments, Dr. Chen, who has served as a director since June 2020, has resigned from the TYRA Board.
  • "On behalf of the Board, I want to thank Isan for his many contributions to TYRA.

Genomenon Partners with Pharming to Advance APDS Diagnosis

Retrieved on: 
Donnerstag, Mai 2, 2024
Mortality, American College, Probability, Laboratory, ANN, Survival, APDS, Disorder, Partnership, Genetic testing, PIK3R1, Molecular diagnostics, Disease, ACMG, Lymphoma, Patient, Bronchiectasis, AMP, Primary immunodeficiency, Caregiver, Genome, Immunodeficiency, Diagnosis, Infection, PIK3CD, Medicine, Activated, Medical genetics, Gene, Medical device

ANN ARBOR, Mich., May 2, 2024 /PRNewswire-PRWeb/ -- Genomenon, a genomic intelligence company, has partnered with Pharming Group in an effort to make variant data on the PIK3CD and PIK3R1 genes available to genetic testing labs, as well as clinicians and researchers, worldwide. This data enables accurate molecular diagnoses and supports the precise medical management of the disease. The partnership also improves the ability of diagnostic laboratories and clinicians to resolve variants of uncertain significance (VUSs) and advances Genomenon's mission to curate the human genome.

Key Points: 
  • APDS, or Activated PI3K delta syndrome, is a rare primary immunodeficiency affecting approximately 1 to 2 people per million worldwide.
  • As the symptoms of APDS can be associated with a variety of conditions, patients typically experience a median 7-year diagnostic delay.
  • "Through our AI-driven technology and genomic expertise, Genomenon has produced the world's most comprehensive variant landscape on PIK3CD and PIK3R1.
  • This curated genetic dataset is being made available to the community through Genomenon's Mastermind Genomic Intelligence Platform to help inform and accelerate the diagnosis for patients with APDS," said Mike Klein, Genomenon CEO.

ASHA-YouGov National Poll: Most Adults With Communication Disorders Report Facing Stigmas

Retrieved on: 
Mittwoch, Mai 1, 2024
Steps, YouGov, American Speech–Language–Hearing Association, Disorder, Stroke, Audiology, Learning, Hearing, Directory, Brain, Language, Communication, CCC, Intelligence, Environment, Social stigma, Stuttering, Marie Cassidy, ASHA, Clutch (eggs), Methodology, Aphasia, Ageing, Quality of life, Organization

ROCKVILLE, Md., May 1, 2024 /PRNewswire/ -- Sixty-five percent of people ages 18 years and older with hearing, speech, and/or language disorders say that they encounter stigmas, according to a new national poll commissioned by the American Speech-Language-Hearing Association (ASHA) and conducted by YouGov. The results are being released as National Speech-Language-Hearing Month begins today.

Key Points: 
  • Although these disorders can make communication more challenging, they do not reflect a person's intelligence.
  • ASHA chose to poll adults because information about the stigmatization that they experience is not as available as it is for children.
  • "It is most fitting that we spotlight stigmas associated with communication disorders for National Speech-Language-Hearing Month," said ASHA President Tena L. McNamara, AuD, CCC-A/SLP.
  • The figures have been weighted and are representative of U.S. adults ages 18+ years with hearing, speech, and/or language difficulties.

HER HIGHNESS SHEIKHA MOZA FORMALLY LAUNCHES QATAR PRECISION HEALTH INSTITUTE

Retrieved on: 
Mittwoch, Mai 1, 2024
Disorder, Genetics, Genomics, Global Alliance, Map, Qatar Genome Programme, Heart, Research, Parent, Qatar Foundation, Environment, Patient, Arab world, Multimedia, Medicine, Antibiotics

DOHA, Qatar, May 1, 2024 /PRNewswire/ -- Her Highness Sheikha Moza bint Nasser, Chairperson of Qatar Foundation, officially launched the Qatar Precision Health Institute, a national body aimed at leveraging the country's advanced genomics research to accelerate personalized healthcare – leading to more precise treatments and improved health outcomes for people in the Arab world and beyond.

Key Points: 
  • DOHA, Qatar, May 1, 2024 /PRNewswire/ -- Her Highness Sheikha Moza bint Nasser, Chairperson of Qatar Foundation, officially launched the Qatar Precision Health Institute, a national body aimed at leveraging the country's advanced genomics research to accelerate personalized healthcare – leading to more precise treatments and improved health outcomes for people in the Arab world and beyond.
  • The Qatar Precision Health Institute builds on the groundbreaking work of Qatar Biobank and Qatar Genome Programme, uniting the efforts of Qatar Foundation's research institutes to provide insights that reveal the effects of lifestyle, environment, and genetics on the Arab population.
  • Her Highness, Sheikha Moza bint Nasser, spoke at the formal launch event, emphasizing the importance of raising public awareness on the benefits of precision health.
  • Qatar Foundation's work in the field of precision health led to the creation of the Qatari genome catalog, comprising more than 88 million variants, 24 million of which had never been reported previously in international databases.