Busulfan

Orca Bio Presents Promising Data on Orca-T in Two Oral Presentations at the 2024 Tandem Meetings of ASTCT® and CIBMTR®

Retrieved on: 
Friday, February 23, 2024
Research, Infectious Diseases, Clinical Trials, Stem Cells, Biotechnology, Health, Pharmaceutical, Science, Oncology, MAC, AML, Syndrome, MD, Assistant, Physician, RFS, NRM, MMF, Weill Cornell Medicine, Risk, Thiotepa, Graft-versus-host disease, Bone marrow, Society, BFT, MDS, CNI, Patient, Hematopoietic stem cell transplantation, NewYork-Presbyterian Hospital, Survival, Transplant, Acute myeloid leukemia, Weill Cornell Medical Center, Cancer, Busulfan, Pharmaceutical industry

In the first of two oral presentations, findings from a retrospective analysis compared historical outcomes between Orca-T and a standard of care allogeneic stem cell transplant (alloHSCT) plus post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis in patients with hematologic malignancies receiving myeloablative conditioning (MAC).

Key Points: 
  • In the first of two oral presentations, findings from a retrospective analysis compared historical outcomes between Orca-T and a standard of care allogeneic stem cell transplant (alloHSCT) plus post-transplant cyclophosphamide (PTCy)-based graft versus host disease (GvHD) prophylaxis in patients with hematologic malignancies receiving myeloablative conditioning (MAC).
  • Within the non-randomized cohorts of varying conditioning regimens and disease types, Orca-T demonstrated favorable results across clinical outcomes including relapse-free survival (RFS), non-relapse mortality (NRM) and overall survival (OS).
  • The analysis included patients with acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) who had a median follow-up time of 15 months and 12 months in the Orca-T and PTCy cohorts, respectively.
  • The outcomes at 12 months are summarized in the table below:

Orchard Therapeutics Announces Agreement with the Beneluxa Consortium Enabling Reimbursed Access to Libmeldy

Retrieved on: 
Thursday, January 25, 2024
Death, Pharmaceutical policy, Patient, MHRA, Therapy, Busulfan, Health care, Civil service commission, European Commission, Health, Mutation, Food, MLD, Central nervous system, Traffic barrier, ARSA, FDA, Priority review, Genome, HSC, Nerve, PDUFA, Orchard, Pharmaceutical industry

BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Jan. 25, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, a global gene therapy leader, today announced the company has reached an agreement with the Beneluxa Initiative on Pharmaceutical Policy (Beneluxa) enabling reimbursed access to Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, approved for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “We are pleased to have reached an agreement with the Beneluxa consortium that enables sustainable access to treatment.
  • Multiple eligible MLD patients are currently in the treatment process and expected to receive Libmeldy in the coming months.

Editas Medicine Announces New EDIT-301 Safety and Efficacy Data in 17 Patients, Presented Today at the American Society of Hematology (ASH) Annual Meeting and in a Company-sponsored Webinar

Retrieved on: 
Monday, December 11, 2023
SCD, Cleveland Clinic, Sickle cell disease, Șaeș, Division, EHA, Editas Medicine, Anemia, Data, Fetal hemoglobin, MD, Society, Clinical trial, Busulfan, Blood, Event, ASH, European Hematology Association, Patient, TDT, Safety, Senior, Pharmaceutical industry

The total dataset of 17 treated patients includes 12 additional patients since the data presentation at the European Hematology Association (EHA) Annual Congress and in a Company-sponsored webinar this past June.

Key Points: 
  • The total dataset of 17 treated patients includes 12 additional patients since the data presentation at the European Hematology Association (EHA) Annual Congress and in a Company-sponsored webinar this past June.
  • Editas Medicine will present the RUBY and EdiTHAL trial data today at 1 p.m.
  • The data will also be presented in a poster presentation at the American Society of Hematology (ASH) Annual Meeting in San Diego, CA, at 6:00 p.m. PT (9:00 p.m.
  • Presenting Author: Rabi Hanna, M.D., Department of Pediatric Hematology Oncology and Blood and Marrow Transplantation, Cleveland Clinic Children’s, Cleveland, OH, United States

Orchard Therapeutics Receives Swissmedic Approval for Libmeldy in Early-onset MLD

Retrieved on: 
Monday, December 11, 2023
Nerve, MLD, Traffic barrier, European Commission, Food, Agency, Priority review, Health care, Death, FDA, PDUFA, Civil service commission, HSC, MHRA, Therapy, Busulfan, ARSA, Mutation, Atidarsagene autotemcel, Genome, Central nervous system, Patient, Health, Pharmaceutical industry

BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).

Key Points: 
  • BOSTON and LONDON, Dec. 11, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the Swiss Agency for Therapeutic Products (Swissmedic) has approved Libmeldy® (atidarsagene autotemcel), a hematopoietic stem cell (HSC) gene therapy, for the treatment of early-onset metachromatic leukodystrophy (MLD).
  • Libmeldy aims to correct the underlying genetic cause of MLD by inserting a working copy of the ARSA gene into the genome of a patients’ own HSCs.
  • With more than a cumulative 250 patient-years of follow-up, Libmeldy was generally well-tolerated, with no treatment-related serious adverse events or deaths.
  • “Today’s approval of Libmeldy in Switzerland opens up tremendous new possibilities for eligible children with MLD who previously had no approved treatment options beyond supportive care,” said Leslie Meltzer, Ph.D., chief medical officer of Orchard Therapeutics.

Long-term Follow-up Data From bluebird’s Gene Therapy Program in Sickle Cell Disease Support Durable, Potentially Curative Benefits Through Stable Production of Anti-Sickling Adult Hemoglobin and Resolution of Vaso-Occlusive Events

Retrieved on: 
Saturday, December 9, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Adolescence, Nasal congestion, Safety, Food, Hemolytic anemia, Sickle cell disease, ASH, Pain, Priapism, Acute myeloid leukemia, Patient, Disease, Acute chest syndrome, Hospital, Quality of life (healthcare), Fatigue, HB, Leukemia, Busulfan, Division, VOE, RAF Sullom Voe, Hemolysis, Myelodysplastic syndrome, Exposition, University, Diagnosis, Society, Quality of life, Biology, Blood transfusion, Bilirubin, Comorbidity, Pharmaceutical industry, Medical imaging, Anemia, Hematology

The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.

Key Points: 
  • The majority of adverse events in treated patients were attributed to underlying sickle cell disease or conditioning with busulfan.
  • Lovo-cel is the most deeply studied gene therapy in development for sickle cell disease, with the most patients treated and longest follow-up in the field.
  • As of February 13, 2023, 59 patients were treated across the entire clinical development program with follow-up beyond 8 years in the earliest treated patients.
  • In sickle cell disease, hemolysis happens too quickly due to the fragility of sickled red blood cells, resulting in hemolytic anemia.

Orca Bio Presents Positive Data Demonstrating the Potential for Orca-T and Orca-Q to Expand Treatment to Additional Patient Groups at the 65th ASH Annual Meeting

Retrieved on: 
Saturday, December 9, 2023
Science, Biotechnology, Research, Pharmaceutical, Oncology, Health, Genetics, Clinical Trials, MAC, Thiotepa, NRM, Safety, Toxicity, CTCAE, Incidence, ASH, Acute myeloid leukemia, Acute leukemia, Syndrome, Risk, University, Mortality, Patient, Busulfan, Infection, BFT, RFS, MDS, Graft-versus-host disease, HSCT, Cancer, Trial of the century, CML, Toxic epidermal necrolysis, Bone marrow, AML, RIC, MD Anderson Cancer Center, Physician, OS, Quality of life, Society, ALL, Face, Pharmaceutical industry, Vaccine, Dentistry, Medical imaging

The results presented at the 65th American Society of Hematology (ASH) Annual Meeting showed promising data with the use of Orca Bio’s cell therapies across several patient groups where there is significant unmet medical need.

Key Points: 
  • The results presented at the 65th American Society of Hematology (ASH) Annual Meeting showed promising data with the use of Orca Bio’s cell therapies across several patient groups where there is significant unmet medical need.
  • Results highlighted in an oral presentation showed Orca-T’s ability to deliver similar outcomes in older patients undergoing myeloablative conditioning (MAC) as younger patients.
  • In this subgroup from the ongoing multi-center Phase 1b clinical trial, Orca-T demonstrated promising results in this patient population (n=16).
  • The estimated incidence of CTCAE grade 2 and greater than grade 3 infections at one year were 9% and 15%, respectively.

 Vertex and CRISPR Therapeutics Announce Authorization of the First CRISPR/Cas9 Gene-Edited Therapy, CASGEVY™ (exagamglogene autotemcel), by the United Kingdom MHRA for the Treatment of Sickle Cell Disease and Transfusion-Dependent Beta Thalassemia

Retrieved on: 
Thursday, November 16, 2023
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Medicine, CRISPR Therapeutics, Therapy, Sickle cell disease, MHRA, Cellular, Innovation, Nobel Prize, Patient, Busulfan, CRSP, Medication, HLA, CRISPR, TDT, Imperial College London, Health care, Volatile organic compound, SCD, Vertex Pharmaceuticals, Pharmaceutical industry, Vertex

Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).

Key Points: 
  • Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) and CRISPR Therapeutics (Nasdaq: CRSP) announced today that the United Kingdom (U.K.) Medicines and Healthcare products Regulatory Agency (MHRA) has granted conditional marketing authorization for CASGEVY™ (exagamglogene autotemcel [exa-cel]), a CRISPR/Cas9 gene-edited therapy, for the treatment of sickle cell disease (SCD) and transfusion-dependent beta thalassemia (TDT).
  • CASGEVY has been authorized for the treatment of eligible patients 12 years of age and older with SCD with recurrent vaso-occlusive crises (VOCs) or TDT, for whom a human leukocyte antigen (HLA) matched related hematopoietic stem cell donor is not available.
  • Once achieved, these benefits are potentially expected to be life-long.
  • The safety profile of 97 SCD and TDT patients treated to date with CASGEVY in these ongoing studies is generally consistent with myeloablative conditioning with busulfan and hematopoietic stem cell transplant.

PrecisePK Redefines Precision Dosing with Academic Integration, Setting New Standards In Therapeutic Drug Monitoring

Retrieved on: 
Wednesday, November 29, 2023
Associate, SAN, Vancomycin, Cardiology, Pharmacist, Patient, Medicine, Busulfan, Neurology, Infection, Tacrolimus, Health, Physician, Networking hardware, Education, Pharmaceutical industry

This acknowledgment fortifies PrecisePK's position as a trusted academic-centric platform revolutionizing precision drug dosing.

Key Points: 
  • This acknowledgment fortifies PrecisePK's position as a trusted academic-centric platform revolutionizing precision drug dosing.
  • This move solidifies PrecisePK's role in advancing therapeutic drug monitoring and precision medicine.
  • This strategic alliance is a pivotal step forward in advancing patient drug safety education and elevating clinical precision drug dosing standards across the region," says Anjum Gupta, CEO of PrecisePK.
  • PrecisePK stands at the forefront of revolutionizing precision dosing with its state-of-the-art Bayesian Model-Informed Precision Dosing (MIPD) software.

Rocket Pharmaceuticals Reports Third Quarter 2023 Financial Results and Highlights Recent Progress

Retrieved on: 
Monday, November 6, 2023
FDA, Health, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, Consensus, Patient, Research, Busulfan, American College, CGMP, Genetic testing, Clinical trial, AAV, PDUFA, Incidence, IMPD, LV, Danon disease, Malignancy, Medication, Mortality, CMC, Conference, CTA, Acquisition, PKP2, Disease, JACC, Journal, Consensus decision-making, Priority review, Male, PKD, Natural history, MHRA, Disorder, EMA, Arrhythmia, Journal of the American College of Cardiology, LAMP2, Safety, Investment, Diagnosis, BLA, Pediatric Blood & Cancer, Pharmaceutical industry, Medical device, Security (finance), Fine chemical, G&A

Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.

Key Points: 
  • Supported peer-reviewed expert consensus paper to increase awareness of diagnosis and clinical management of patients with Danon Disease.
  • Highlights of the publication include a review of diagnosing Danon Disease emphasizing the importance of genetic testing upon clinical suspicion, natural history, management recommendations and recent advances in potential gene therapy treatment.
  • Rocket is working towards initiation of Phase 2 pivotal trial activities in Europe and the UK.
  • As of September 30, 2023, Rocket had cash, cash equivalents and investments of $437.2 million.

bluebird bio to Present New and Updated Data from Gene Therapy Programs in Sickle Cell Disease and Beta-Thalassemia at the 65th American Society of Hematology (ASH) Annual Meeting and Exposition

Retrieved on: 
Thursday, November 2, 2023
Health, Genetics, Other Health, General Health, Pharmaceutical, Biotechnology, Hemoglobinopathy, FDA, MSCE, PDUFA, Busulfan, Food, LVV, SCD, History, Sickle cell disease, Ronas Voe, Prescription Drug User Fee Act, Data, Children's Hospital of Philadelphia, Thalassemia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, TDT, Max Healthcare, University of Pennsylvania, Pediatrics, Society, ASH, Priority review, Quality of life, San Diego Convention Center, Division, HRQOL, Perelman School of Medicine at the University of Pennsylvania, University, Exposition, Month, Hospital, BLA, Betibeglogene autotemcel, Pharmaceutical industry, Vaccine, Patient, Quality of life (healthcare), Safety, Hematology

The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.

Key Points: 
  • The meeting will take place December 9-12, 2023 at the San Diego Convention Center and online.
  • lovo-cel treatment regimen largely reflects known side effects of hematopoietic stem cell collection and busulfan conditioning regimen and underlying sickle cell disease.
  • Updated iron management outcomes demonstrating sustained improvements in iron burden, with the majority of patients able to stop iron chelation therapy, will also be presented.
  • Safety of beti-cel treatment largely reflects the known side effects of hematopoietic stem cell collection and busulfan conditioning regimen.