Phenotype

Atara Biotherapeutics Announces First Quarter 2024 Financial Results and Operational Progress

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木曜日, 5月 9, 2024
Stem Cells, Biotechnology, Health, General Health, Pharmaceutical, Oncology, Research, Infectious Diseases, Genetics, Science, Clinical Trials, Mantle cell lymphoma, Cancer, International Society, Investment, Cell, Phenotype, Safety, Risk, Pharmacokinetics, Poster, Lymphoproliferative disorders, Depreciation, Cytokine, Partnership, Lymphoma, Biologics license application, ATRA, Follicular lymphoma, CD19, ASH, Epstein–Barr virus, Patient, NHL, LD, Toxicity, BLA, Memory, Workforce, EBV, Biomarker, SLE, Tabelecleucel, Pharmaceutical industry

Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today reported financial results for the first quarter 2024, recent business highlights, and key upcoming milestones for 2024.

Key Points: 
  • Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today reported financial results for the first quarter 2024, recent business highlights, and key upcoming milestones for 2024.
  • “This is anticipated to provide multiple near-term clinical milestones for ATA3219, including initial non-Hodgkin’s lymphoma data expected in the fourth quarter 2024, and initial lupus nephritis data in the first half of 2025, with plans to expand into a new SLE cohort without lymphodepletion.
  • Subjects will receive lymphodepletion treatment followed by ATA3219 at a dose of 40, 80, or 160 x 106 CAR+ T cells.
  • Subjects will receive LD treatment followed by ATA3219 at a dose of 40, 80, 240, or 480 x 106 CAR+ T cells.

Fate Therapeutics Announces First Lupus Patient Treated in Phase 1 Autoimmunity Study of Off-the-shelf FT819 CAR T-cell Program

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木曜日, 5月 9, 2024
FDA, University, Cancer, Periphery, Phenotype, Safety, CXCR4, Pharmacokinetics, Risk, Food, Destiny, Cell, B cell, Lupus, Nebraska Medicine, IND, Autoimmunity, Investigational New Drug, CD19, ADR, Patient, Principal, Gene, Society, IPSC, BCL, BCM, Memory, Hospital, Immune system, SLE, Therapy, Lymphoid leukemia, B-cell lymphoma, Vaccine

SAN DIEGO, May 09, 2024 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune diseases, today announced that the first patient with systemic lupus erythematosus (SLE) has been treated in the Phase 1 autoimmunity study of FT819, the Company’s off-the-shelf, CD19-targeted chimeric antigen receptor (CAR) T-cell program. In addition, at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, the Company today presented translational data from the Phase 1 study of FT819 in relapsed / refractory B-cell malignancies (BCM) and initial clinical observations from the Phase 1 study of its FT522 off-the-shelf, CD19-targeted CAR NK cell program in relapsed / refractory B-cell lymphoma (BCL). Data from these programs highlight the scientific rationale and demonstrate key therapeutic mechanisms of activity for the treatment of B cell-mediated autoimmune disease.

Key Points: 
  • Data from these programs highlight the scientific rationale and demonstrate key therapeutic mechanisms of activity for the treatment of B cell-mediated autoimmune disease.
  • The multi-center, Phase 1 autoimmunity study of FT819 is designed to assess safety, pharmacokinetics, and anti-B cell activity for patients with moderate-to-severe SLE (NCT06308978).
  • The first patient, a 27 year-old woman diagnosed with SLE over ten years ago who has refractory disease despite having been treated with multiple standard-of-care therapies, received conditioning chemotherapy followed by a single dose of FT819 at 360 million cells.
  • The patient was discharged after a three-day hospital stay without any notable adverse events.

Vor Bio Reports First Quarter 2024 Financial Results and Provides Company Update

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木曜日, 5月 9, 2024
CD33, Patient, Phenotype, AML, Administration, Transplant, Research and development, Bone marrow, Acute myeloid leukemia, Lymphocyte, Clinical trial, MDS, G&A

CAMBRIDGE, Mass., May 09, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period ended March 31, 2024, and provided a business update.

Key Points: 
  • CAMBRIDGE, Mass., May 09, 2024 (GLOBE NEWSWIRE) -- Vor Bio (Nasdaq: VOR), a clinical-stage cell and genome engineering company, today reported financial results for the three-month period ended March 31, 2024, and provided a business update.
  • Research & Development (R&D) Expenses: R&D expenses for the first quarter of 2024 were $24.3 million, compared to $21.9 million for the first quarter of 2023.
  • General & Administrative (G&A) Expenses: G&A expenses for the first quarter of 2024 were $8.0 million, compared to $8.5 million for the first quarter of 2023.
  • Net Loss: Net loss for the first quarter of 2024 was $30.8 million, compared to $28.4 million for the first quarter of 2023.

Capsida Biotherapeutics Presents New Preclinical Evidence Indicating Novel First-in-Class IV-Administered Gene Therapy Effectively Treats Genetic Epilepsy Due to STXBP1 Mutations

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火曜日, 5月 7, 2024
Doctor of Philosophy, Brain, Traffic barrier, Mouse, Phenotype, Poster, MD, Model, Cell, Deficit spending, Associate professor, STXBP1, AAV, DSM-IV codes, Vivisection, Genetics, Disease, Patient, Liver, Baylor College of Medicine, Society, Gene, Treatment, Neurology, Seizure, Gene expression, Neuron, Annual general meeting, Texas Children's Hospital, Baylor University, Baltimore Convention Center, Vaccine

THOUSAND OAKS, Calif., May 7, 2024 /PRNewswire/ -- Capsida Biotherapeutics ("Capsida") today announced new preclinical data supporting the potential of Capsida's gene therapy candidate, CAP-002, to achieve levels of gene supplementation necessary to correct neurological phenotypes associated with genetic epilepsy due to syntaxin-binding protein 1 (STXBP1) mutations.

Key Points: 
  • Gene therapy for genetic epilepsy due to STXBP1 mutations has not been previously possible because earlier generation adeno-associated viruses (AAVs) or wild-type AAVs could not achieve the level of widespread neuronal transduction required to modify the disease.
  • CAP-002 is a first-in-class next-generation intravenous (IV)-administered gene therapy that achieves brain-wide neuronal expression while simultaneously detargeting the liver.
  • Capsida's wholly owned program is currently in IND-enabling studies and is expected to enter the clinic in the first half of 2025.
  • This level of gene expression raises neuronal STXBP1 protein to levels comparable to those that reversed disease phenotype in the mouse model.

Werewolf Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Update

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金曜日, 5月 3, 2024
Cancer, Irritable bowel syndrome, Phenotype, IL-2, Food, Research, Pembrolizumab, Colitis, Immunotherapy, PacWest Bancorp, Tumor microenvironment, Defecation, ASCO, Therapy, Immunology, Patient, Society, BPR, Poster, Clinical trial, Annual general meeting, Non-Hodgkin lymphoma, Food and Drug Administration, DSM-IV codes, Pharmaceutical industry

“Werewolf continues to focus on the execution of our lead clinical programs WTX-124 and WTX-330, with updates planned for later in the second quarter.

Key Points: 
  • “Werewolf continues to focus on the execution of our lead clinical programs WTX-124 and WTX-330, with updates planned for later in the second quarter.
  • Financial Results for the First Quarter of 2024:
    Cash position: As of March 31, 2024, cash and cash equivalents were $139.2 million, compared to $134.3 million as of December 31, 2023.
  • General and administrative expenses: General and administrative expenses were $5.0 million for each of the first quarters of 2024 and 2023.
  • Net loss: Net loss was $16.2 million for the first quarter of 2024, compared to $12.0 million for the same period in 2023.

AUA, SUFU Release Guideline on Diagnosis and Treatment of Overactive Bladder

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火曜日, 4月 23, 2024
Society, Phenotype, Woman, American Urological Association, MD, Partnership, Pelvic floor dysfunction, Guideline, OAB, Obesity, Patient, Overactive bladder, AUA, Constipation, Diagnosis, SUFU, Quality of life, Prevalence, Systematic review, Medical device

BALTIMORE, April 23, 2024 (GLOBE NEWSWIRE) -- Today, the American Urological Association (AUA), in partnership with the Society of Urodynamics, Female Pelvic Medicine & Urogenital Reconstruction (SUFU), released the 2024 clinical practice guideline for the diagnosis and treatment of overactive bladder (OAB).

Key Points: 
  • BALTIMORE, April 23, 2024 (GLOBE NEWSWIRE) -- Today, the American Urological Association (AUA), in partnership with the Society of Urodynamics, Female Pelvic Medicine & Urogenital Reconstruction (SUFU), released the 2024 clinical practice guideline for the diagnosis and treatment of overactive bladder (OAB).
  • As OAB is a symptom-based diagnosis, the impact of the symptoms on quality of life is critical to the condition.
  • The degree of bother caused by OAB symptoms directly affects OAB care-seeking, treatment intensity, and satisfaction with treatment.
  • The AUA/SUFU guideline on the diagnosis and treatment of idiopathic overactive bladder.

Voyager Therapeutics to Present Broad Set of Translational Data Supporting IV-Delivered, CNS Gene Therapy Programs Advancing Toward Clinical Trials at the ASGCT 27th Annual Meeting

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月曜日, 4月 22, 2024
TRACER, Histology, Mouse, Phenotype, Data science, Cell, AAV, CNS, Machine learning, Senior, Tauopathy, Gene, Process, Society, BBB, ALS, Central nervous system, Public, HEK293, Therapy, Centrifuge, SOD1, Vaccine

“Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.

Key Points: 
  • “Voyager’s novel TRACER-derived capsids underlie 13 partnered programs and three wholly-owned programs to enable IV-delivery of gene therapies for diseases of the central nervous system.
  • Three of those programs now have development candidates selected, and we see the potential for them to enter clinical trials next year,” said Todd Carter, Ph.D., Chief Scientific Officer of Voyager Therapeutics.
  • ET
    Intravenous administration of BBB-penetrant, MAPT-Silencing, AAV gene therapy provides broad and robust CNS Tau lowering in tauopathy mouse models (#1602).
  • ET
    Intravenous delivery of AAV gene therapy for the treatment of SOD1-ALS provides broad SOD1 lowering in NHP (#1647).

Lexeo Therapeutics Announces License Agreement to Accelerate Development of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

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月曜日, 4月 22, 2024
FDA, Weill Cornell Medicine, Patient, Fast Track, Phenotype, Death, Cardiomyopathy, Research, Natural history, IND, Cornell University, Senior, Friedreich's ataxia, Cardiovascular disease, Therapy, Orphan drug, Pharmaceutical industry

NEW YORK, April 22, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company, today announced an in-license agreement with Cornell University to expedite development of the investigational gene therapy candidate LX2006 for the treatment of Friedreich ataxia (FA) cardiomyopathy.

Key Points: 
  • Under the license agreement, Lexeo has acquired certain rights1 including rights to current and future data generated in an ongoing investigator-initiated Phase 1A trial of AAVrh.10hFXN to treat FA cardiomyopathy ( NCT05302271 ).
  • The agreement will support Lexeo’s efforts to develop a potentially life-changing therapy for this unmet need.
  • Lexeo previously licensed know-how relating to AAVrh.10hFXN from Weill Cornell Medicine and collaborated with researchers there to further study the candidate, which Lexeo refers to as LX2006.
  • “This agreement with Lexeo Therapeutics builds upon years of collaboration between Weill Cornell Medicine and Lexeo to benefit patients with FA cardiomyopathy.

GeneFab Announces Two Presentations at the American Society of Gene & Cell Therapy (ASGCT) Annual Meeting 2024

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月曜日, 4月 22, 2024
FDA, Phenotype, Machine learning, Cell, Protein engineering, Immunotherapy, OMICS, Gene, Society, Synthetic biology, Macrophage, Gene expression

ALAMEDA, Calif., April 22, 2024 (GLOBE NEWSWIRE) -- GeneFab, LLC (“GeneFab”), an expert contract research, development and manufacturing organization (CRDMO) today announced two poster presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which will be held May 7 to 11, 2024 in Baltimore, MD.

Key Points: 
  • ALAMEDA, Calif., April 22, 2024 (GLOBE NEWSWIRE) -- GeneFab, LLC (“GeneFab”), an expert contract research, development and manufacturing organization (CRDMO) today announced two poster presentations at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, which will be held May 7 to 11, 2024 in Baltimore, MD.
  • GeneFab employs expert genetic design and manufacturing methods to meet Advanced Therapy companies' needs.
  • With a 92,000 square foot cGMP manufacturing facility in Alameda, California that supports various customer projects, including allogeneic cell therapy, CAR-T and synthetic biology research services.
  • GeneFab’s presentations highlight two new technologies for control of payload production from cell therapies.

n-Lorem Foundation Collaborates with Across Healthcare to Develop a Custom Submission Platform for Physicians of Nano-rare Patients

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火曜日, 4月 16, 2024
Technology, Health, Philanthropy, General Health, Pharmaceutical, Health Technology, Other Science, Research, Software, Foundation, Science, Mutation, Phenotype, Patient, Access, Physician, Caregiver, Pharmaceutical industry

n-Lorem, a nonprofit Foundation , has collaborated with Across Healthcare, a technology innovator, to develop a first-of-its-kind submission platform and system that allows research physicians from across the United States to submit applications for treatment for their patients with nano-rare illnesses.

Key Points: 
  • n-Lorem, a nonprofit Foundation , has collaborated with Across Healthcare, a technology innovator, to develop a first-of-its-kind submission platform and system that allows research physicians from across the United States to submit applications for treatment for their patients with nano-rare illnesses.
  • “The n-Lorem team performs a comprehensive evaluation for every individual affected by a nano-rare mutation seeking treatment through our application platform.
  • The user-friendly, custom platform enables each physician to create a personal account to be able to submit and track the progress of multiple applications.
  • “When n-Lorem approached us about their unique platform needs, we knew that we had to help,” said Jason Colquitt, CEO, Across Healthcare.