SCID

TeleRare Health™ Announces Launch of National Virtual Clinic for Rare Disease Patients

Retrieved on: 
Tuesday, April 30, 2024
Wilson's disease, Bangladesh Technical Education Board, Genomics, Kidney, SCID, Diagnosis, Safety, NIH, AI, Heart, Adrenoleukodystrophy, MD, Partnership, Children's National Hospital, DMD, FXS, Research, Human, Genetic testing, Disease, Patient, ALD, Pediatrics, Krabbe disease, Duchenne muscular dystrophy, PD, GMG, Fragile X syndrome, Pharmaceutical industry

MINNEAPOLIS, April 30, 2024 /PRNewswire/ -- 8:00a (CET) April 30, 2024 - TeleRare Health™ launched today, a virtual clinic for Rare Disease patients and their families.

Key Points: 
  • MINNEAPOLIS, April 30, 2024 /PRNewswire/ -- 8:00a (CET) April 30, 2024 - TeleRare Health™ launched today, a virtual clinic for Rare Disease patients and their families.
  • TeleRare Health aims to be the premier provider of virtual care for rare and genomic disease nationwide, envisioning a world where all patients have access to cutting edge diagnostics, treatments, and specialists.
  • "Significant breakthroughs have occurred in the management of rare disease," said Alex Katz, MD, medical geneticist and Chief Medical Officer of TeleRare Health.
  • We're overdue for a virtual clinic for the millions of Americans with rare disease conditions, and TeleRare Health's launch is cause for optimism.

Revvity Launches EONIS Q System Enabling Faster, Simplified Newborn Screening for SMA and SCID

Retrieved on: 
Monday, November 27, 2023
Technology, Biotechnology, Health, Neurology, Consumer, Pharmaceutical, Stem Cells, Other Science, Medical Devices, Software, Managed Care, Baby, Maternity, Health Technology, Science, Infant, European Alliance, Disease, Birth, PCR, Heart, Laboratory, CE, Workflow, Child, SCID, SMA, Real-time, Life, Pharmaceutical industry

The EONIS Q system simplifies and streamlines molecular testing for SMA and SCID with an innovative workflow, inclusive of the EONIS™ Q96 instrument, the EONIS™ SCID-SMA kit and dedicated EONIS™ EASI™ software.

Key Points: 
  • The EONIS Q system simplifies and streamlines molecular testing for SMA and SCID with an innovative workflow, inclusive of the EONIS™ Q96 instrument, the EONIS™ SCID-SMA kit and dedicated EONIS™ EASI™ software.
  • Compared to other methods, the EONIS Q system uses fewer consumables and one-time use plasticware, further optimizing resource and cost efficiencies for labs looking to adopt the system.
  • The European Alliance for Newborn Screening in Spinal Muscular Atrophy (“SMA”), an advocacy group established by SMA Europe, mandates that by 2025, all newborns in Europe should be screened for SMA.
  • The CE-IVD declaration of the EONIS Q system and other solutions like it advance progress toward achieving this goal.

Cidara Therapeutics Presents New Preclinical and Clinical Data on Novel Drug-Fc Conjugate CD388 at IDWeek 2023

Retrieved on: 
Wednesday, October 11, 2023
Pharmacokinetics, DFC, SCID, Universal, CDTX, Janssen, Johnson & Johnson, Tavarua, Safety, Drug, Boston Convention and Exhibition Center, Therapy, Doctor of Philosophy, SAN, Janssen Pharmaceuticals, Convention center, Intramuscular injection, Patient, Pharmaceutical industry, Vaccine

SAN DIEGO, Oct. 11, 2023 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) therapies designed to save lives and improve the standard of care for patients facing serious diseases, today announced new data on its drug-Fc conjugate (DFC) candidate, CD388, at IDWeek 2023.

Key Points: 
  • SAN DIEGO, Oct. 11, 2023 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) therapies designed to save lives and improve the standard of care for patients facing serious diseases, today announced new data on its drug-Fc conjugate (DFC) candidate, CD388, at IDWeek 2023.
  • The conference is taking place both in-person at the Boston Convention and Exhibition Center in Boston, MA and virtually from October 11 – 15, 2023.
  • “It is important to highlight these new data demonstrating the safety, efficacy and tolerability of CD388 in preventing influenza A and B,” said Jeffrey Stein, Ph.D. president and chief executive officer of Cidara.
  • Janssen recently delivered its Election to Proceed Notice for CD388 with the intent to transfer its rights and obligations to another entity.

Cidara Therapeutics to Present New Data on Novel drug-Fc conjugate CD388 at IDWeek 2023

Retrieved on: 
Wednesday, September 27, 2023
Drug, SCID, Intramuscular injection, Boston Convention and Exhibition Center, Janssen, CDTX, Janssen Pharmaceuticals, DFC, Patient, Pharmacokinetics, SAN, Convention center, Johnson & Johnson, Universal, Therapy, Pharmaceutical industry, Vaccine

SAN DIEGO, Sept. 27, 2023 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, today announced that it will present new data on its drug-Fc conjugate (DFC) candidate, CD388, in an oral presentation and two poster presentations at IDWeek 2023.

Key Points: 
  • SAN DIEGO, Sept. 27, 2023 (GLOBE NEWSWIRE) -- Cidara Therapeutics, Inc. (Nasdaq: CDTX), a biotechnology company using its proprietary Cloudbreak® platform to develop drug-Fc conjugate (DFC) immunotherapies designed to save lives and improve the standard of care for patients facing serious diseases, today announced that it will present new data on its drug-Fc conjugate (DFC) candidate, CD388, in an oral presentation and two poster presentations at IDWeek 2023.
  • The conference will take place both in-person at the Boston Convention and Exhibition Center in Boston, MA and virtually from October 11 – 15, 2023.
  • CD388 is being developed for the universal protection of influenza A and B under an exclusive worldwide collaboration agreement with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company.
  • Janssen recently delivered its Election to Proceed Notice for CD388 with the intent to transfer its rights and obligations to another entity.

EveryLife Foundation Study Measures Economic Impact of Delayed Diagnosis of Rare Diseases

Retrieved on: 
Thursday, September 14, 2023
Research, Congressional News, Views, Public Policy, Government, Healthcare Reform, Health, State, Local, General Health, Other Science, Science, Bureau of Labor Statistics, Workforce, Medicine, Medicaid, Life, New Era, ALD, Bureau, Survival, Quality of life, Pfizer, Glycogen storage disease type II, Pharmacy, Anatomical pathology, Diagnosis, Patient engagement in Canada, Occupational therapy, Genetic counseling, FXS, Genetic testing, UnitedHealth Group, Pharmaceutical Research and Manufacturers of America, Sibling, DMD, Alexion Pharmaceuticals, HIV disease progression rates, American College of Medical Genetics and Genomics, Patient, Pediatrics, Physician, Clutch (eggs), Sanofi, Rare, Amicus Therapeutics, Mortality, Infant, American College, Medical genetics, Disease, Medical imaging, Medical device, Pharmaceutical industry, SCID, Genomics, Genentech, Ultragenyx, Optum, Policy

This new study on the Cost of Delayed Diagnosis is a follow-up to the landmark 2021 “ National Economic Burden of Rare Disease Study ,” which estimated that the economic impact of 379 rare diseases in 2019 was nearly $1 trillion, with 60% of those costs being shouldered directly by families and society.

Key Points: 
  • This new study on the Cost of Delayed Diagnosis is a follow-up to the landmark 2021 “ National Economic Burden of Rare Disease Study ,” which estimated that the economic impact of 379 rare diseases in 2019 was nearly $1 trillion, with 60% of those costs being shouldered directly by families and society.
  • “Medical costs for rare diseases are inevitable, but avoidable costs from delayed diagnosis not only place financial strain on individuals and families but also divert crucial healthcare funds.
  • For individuals with late onset Pompe disease, delayed diagnosis increased mean annual direct medical costs in the year of diagnosis by approximately $50,000 per patient compared with timely diagnosis.
  • For more information about “Cost Benefits of Early Diagnosis and Screening in Rare Disease” study, visit the study website at everylifefoundation.org/delayed-diagnosis-study .

Perspective Therapeutics Presents Positive Preclinical Alpha Particle Therapy Data at the World Molecular Imaging Congress 2023 and the 36th Annual Congress of the European Association of Nuclear Medicine

Retrieved on: 
Monday, September 11, 2023
Survival rate, Glomus tumor, NETS, Radiation, Index, Probability, Metastasis, EANM, SCID, Neoplasm, Survival, WWIC, Toxicity, Research, Nuclear medicine, Neuroendocrine cell, Congress, Korea Military Academy, Korea, CATX, WMIC, Mouse, MTD, European Association for the Study of the Liver, Therapy, NYSE, Human, Pharmaceutical industry, Medical imaging

SEATTLE, WA, Sept. 11, 2023 (GLOBE NEWSWIRE) -- Perspective Therapeutics, Inc. (“Perspective” or “the Company”) (NYSE AMERICAN: CATX), today announced the presentation of preclinical data from two independent studies at the World Molecular Imaging Congress (WMIC) 2023, held on September 5-9, 2023, in Prague, Czech Republic, and the 36th Annual Congress of the European Association of Nuclear Medicine (EANM) in Vienna, Austria, from September 9-13, 2023.

Key Points: 
  • Dr. Michael K. Schultz, Chief Scientific Officer at Perspective Therapeutics, presented research at the WWIC on the potential of lead-based targeted alpha particle therapy using [212Pb]VMT-α-NET for the treatment of neuroendocrine tumors (NETs).
  • The study was carried out by Dr. Dongyoul Lee, an assistant professor at the Korea Military Academy.
  • The dose depositions in tumor and normal organs were estimated based on 203Pb biodistribution data.
  • In addition, tumor progression, survival rates, and body weight changes over a 120-day period were assessed, and favorable radiochemical characteristics were observed.

Altogen Labs Validates a Comprehensive Range of Lung Cancer Xenograft Animal Models for Cutting-Edge Oncology Research

Retrieved on: 
Friday, August 25, 2023
Human, Therapy, Genetics, CDX, SCID, Cancer, Lung cancer, PDX, Cancer cell, Patient, Vivo, Mouse, Diagnosis, NCI, Metastasis, Safety, Pharmaceutical industry, Medical imaging, Calu-3

AUSTIN, Texas, Aug. 25, 2023 /PRNewswire/ -- Altogen Labs, a preclinical CRO laboratory, has announced the validation of a comprehensive array of 10 lung cancer xenograft models.

Key Points: 
  • AUSTIN, Texas, Aug. 25, 2023 /PRNewswire/ -- Altogen Labs, a preclinical CRO laboratory, has announced the validation of a comprehensive array of 10 lung cancer xenograft models.
  • An array of lung cancer xenograft models can significantly improve the chances of a new lung cancer therapy being successful in humans, fewer therapies will fail in clinical trials, and more effective and less toxic lung cancer drugs will be developed.
  • Altogen Labs offers several PDX models and 10 in-house validated lung cancer xenograft models, including A549 , H460 , H226 , NCI-H1975 , DMS273 , LL/2 , Calu-3 , Calu-6 , H1155 , and NCI-H522 .
  • Altogen Labs array of lung cancer xenograft models provides a rich platform to capture the heterogeneity of lung cancer, reflecting its morphology, genetics, and stage-specific phenotypes.

CHOP and Penn Medicine Researchers Develop "In Vivo" RNA-based Gene Editing Model for Blood Disorders

Retrieved on: 
Thursday, July 27, 2023
Nanoparticle, Bone marrow, Doctor of Philosophy, Bangladesh Technical Education Board, COVID-19 vaccine, MD, Patient, Division, Risk, Thomas, Perelman School of Medicine at the University of Pennsylvania, LNP Media Group, CD117, DSM-IV codes, Infection, Disease, Children's Hospital of Philadelphia, Tissue, Hematologic disease, BS, University, Pediatrics, Therapy, CHOP, Hematology, Gene editing, Biomedical Research, Mutation, Research, Penn Institute for Urban Research, DOI, Hospital, HSC, NIH, Vaccine, Hematopoietic stem cell, Stem cell, COVID-19, Philadelphia, Cell, Child, Antibody, Messenger, Blood, Immune system, LNP, Radiation, Doctor of Pharmacy, Cell death, PUMA, SCID, Sickle cell disease, RNA, Penn, Health, Dicarboxylic acid, Dentistry, Pharmaceutical industry, Science

PHILADELPHIA, July 27, 2023 /PRNewswire/ -- In a step forward in the development of genetic medicines, researchers at Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders, allowing for the modification of diseased blood cells directly within the body. If translated into the clinic, this approach could expand access and reduce the cost of gene therapies for blood disorders, many of which currently require patients receive chemotherapy and a stem cell transplant. The findings were published today in the journal Science.

Key Points: 
  • This approach could expand access and reduce the cost of gene therapies for blood disorders.
  • A targeted mRNA-encoded genomic editing methodology could lead to controlled expression, high editing efficacy, and potentially safer in vivo genomic modification compared to currently available technologies."
  • In patients with non-malignant hematopoietic disorders like sickle cell disease and immunodeficiency disorders, these blood cells don't function correctly because they carry a genetic mutation.
  • First, the researchers tested CD117/LNP encapsulating reporter mRNA to show successful in vivo mRNA expression and gene editing.

Homology Medicines Announces First Data from IND-Enabling Studies with GTx-mAb Candidate HMI-104 for PNH, Which Demonstrated Sustained Expression of Functional C5mAb Levels, at the ASGCT Annual Meeting

Retrieved on: 
Tuesday, May 16, 2023
Inhibitor, Neutralizing antibody, Hemolysis, Gene, NOD, MLD, PAH, Serum, SCID, Phenylketonuria, Trabecular meshwork, Gene editing, Gene expression, FIX, Publication, Metachromatic leukodystrophy, Messenger, PKU, Future, Administration, Immunosuppression, Genome, Intravitreal administration, Homologous recombination, Treatment, Vivisection, Immune system, PNH, Cell, Retinal pigment epithelium, NHP, Poster, RPE, Detection, Identification, Virus, ARSA, Society, Vaccine, Pharmaceutical industry, Truffle, Mouse, IND, C5

BEDFORD, Mass., May 16, 2023 (GLOBE NEWSWIRE) -- Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines company, announced today six presentations supporting its gene editing, gene therapy and GTx-mAb programs at the American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, including the first data from IND-enabling studies with HMI-104, the anti-C5 GTx-mAb development candidate for paroxysmal nocturnal hemoglobinuria (PNH). These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.

Key Points: 
  • These preclinical data demonstrated that a one-time administration of HMI-104 resulted in sustained expression of C5 monoclonal antibody (C5mAb) levels, supporting the use of a one-time vectorized approach for PNH to enable continuous antibody production.
  • Additionally, Homology highlighted methods to identify genomic sites with improved homologous-recombination (HR)-based gene editing integration, which could be used to enhance and streamline development of future product candidates.
  • These data support the immunosuppression regimen used in Homology’s ongoing gene editing and gene therapy clinical trials.
  • In the poster, “Gene Therapy Candidate for Metachromatic Leukodystrophy (MLD): Optimization of HMI-202 Leading to HMI-204 Nomination,” Homology highlighted preclinical data on its optimized HMI-204 gene therapy candidate for MLD.

Jasper Therapeutics Appoints David Hinds as Senior Vice President, Development Operations

Retrieved on: 
Monday, April 10, 2023
Citadel, DevOps, University, Military, SCID, The Citadel, Sutro Biopharma, ICON, General partnership, University of South Carolina, Fanconi anemia, Master of Business Administration, MDS, Business administration, Darla Moore School of Business, Hardpan, Rituximab, CD117, ICON PLC, Therapy, Master, Pharmaceutical industry, Science, Takeda, Pirfenidone, Biology, Celecoxib, Jasper

REDWOOD CITY, Calif., April 10, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria, lower to intermediate risk myelodysplastic syndromes (MDS) as well as novel stem cell transplant conditioning regimes, today announced the appointment of David Hinds, as the Company’s Senior Vice President, Development Operations.

Key Points: 
  • REDWOOD CITY, Calif., April 10, 2023 (GLOBE NEWSWIRE) -- Jasper Therapeutics, Inc. (Nasdaq: JSPR) (Jasper), a biotechnology company focused on development of briquilimab, a novel antibody therapy targeting c-Kit (CD117) to address diseases such as chronic spontaneous urticaria, lower to intermediate risk myelodysplastic syndromes (MDS) as well as novel stem cell transplant conditioning regimes, today announced the appointment of David Hinds, as the Company’s Senior Vice President, Development Operations.
  • His outstanding track record in setting strategic directions for clinical research and operations will allow us to effectively advance our development programs for briquilimab,” said Ronald Martell, President and Chief Executive Officer of Jasper.
  • Previously he served as Vice President, Clinical Operations at PellePharm and Tioma Therapeutics.
  • He also led the clinical development and operations strategy at Alios Biopharma, Takeda, Clinipace, Exelixis, Kai Pharmaceuticals, Genentech, Genitope Corporation, and Intermune.