HSCT

Interim Management Statement Q1 2024 of Molecular Partners: Pipeline Progressing with Two Additional Programs to Enter the Clinic in 2025, Update to MP0533 Program

Retrieved on: 
torsdag, maj 16, 2024
Partnership, Molecular imaging, Delta, Novartis, HSC, IND, Bone marrow, Cytokine release syndrome, Congress, AML, CD70, EHA, Hematopoietic stem cell transplantation, Depreciation, Therapy, District court, Molecule, CD33, Conference, CD47, Society, Annual, Alpha particle, B cell, Physician, Half-life, DARPin, ASH, DRS, Stem cell, Molecular Partners, Safety, Tetra, Gene family, ASCO, FAP, Radionuclide, DLL3, CD40, CHF, European Hematology Association, Antigen, Patient, Prejudice, HSCT, SNMMI, Partner, RDT, ELN, CD123, DSM-IV codes, Pharmaceutical industry

For our emerging pipeline, we plan to announce preclinical data from our Switch-DARPin Platform at EHA and anticipate translational efficacy data in the second half of 2024.

Key Points: 
  • For our emerging pipeline, we plan to announce preclinical data from our Switch-DARPin Platform at EHA and anticipate translational efficacy data in the second half of 2024.
  • Of this figure, approximately CHF 8 million will be non-cash effective costs for share-based payments, IFRS pension accounting and depreciation.
  • With CHF 174.1 million in cash and short-term time deposits and no debt as of March 31, 2024, the Company expects to be funded well into 2026.
  • The first Switch-DARPin program (cKIT x CD16a x CD47) was introduced at the annual J.P. Morgan Healthcare Conference in January 2024.

Akari Therapeutics Reports First Quarter 2024 Financial Results and Recent Highlights

Retrieved on: 
torsdag, maj 16, 2024
Security (finance), HSCT, Therapy, Optimism, ADC, MD, M.D, Interim, Geographic atrophy, Research

Entered into a Definitive Merger Agreement with Peak Bio; Expected to Close in the Third Quarter of 2024

Key Points: 
  • Entered into a Definitive Merger Agreement with Peak Bio; Expected to Close in the Third Quarter of 2024
    BOSTON and LONDON, May 16, 2024 (GLOBE NEWSWIRE) -- Akari Therapeutics, Plc (Nasdaq: AKTX), a late-stage biotechnology company developing advanced therapies for autoimmune and inflammatory diseases, has reported financial results for the first quarter ended March 31, 2024 as well as recent company highlights.
  • “My first few weeks as Interim CEO of Akari have been filled with non-stop activity and optimism,” said Samir R. Patel, MD, Akari Interim President and CEO.
  • The combined entity will operate as Akari Therapeutics, Plc, which is expected to continue to be listed and trade on the Nasdaq Capital Market as AKTX.
  • The merger is expected to close in the third quarter of 2024.

Rocket Pharmaceuticals Presents Positive Data from LV Hematology Portfolio at the 27th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT)

Retrieved on: 
fredag, maj 10, 2024
Research, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, Oncology, PKD, Fanconi anemia, Patient, Cell, Survival, HSCT, FA, Disorder, Marketing, Food, European Medicines Agency, FDA, Medication, Safety, BMF, Bone marrow, PDUFA, Infection, Hospital, Gene, EMA, Hemolysis, Biologics license application, BLA, Society, MAA, Busulfan, Pyruvate kinase deficiency, Graft-versus-host disease, Quality of life, Pharmaceutical industry

“The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals.

Key Points: 
  • “The positive updates presented at this year’s annual meeting demonstrate the sustained safety and efficacy across the totality of our LV hematology portfolio,” said Jonathan Schwartz, M.D., Chief Medical & Gene Therapy Officer, Rocket Pharmaceuticals.
  • In our pivotal studies of RP-L102 for Fanconi Anemia, we continue to see maintained genetic and phenotypic correction combined with hematologic stabilization.
  • Additionally, our Phase 1 study of RP-L301 for PKD shows sustained clinically meaningful hemoglobin improvement in all patients.
  • The oral presentation includes positive, updated data (cut-off September 11, 2023) from the global Phase 1/2 pivotal studies of RP-L102, Rocket’s ex vivo LV gene therapy candidate for FA.

Seres Therapeutics Reports First Quarter 2024 Financial Results and Provides Business Updates

Retrieved on: 
onsdag, maj 8, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Oaktree Capital Management, Sale, FDA, Variometer, Gastroenterology, David Berry (inventor), Acceleration, Safety, Clostridioides, Risk, Commercial, Research, Chronic liver disease, Recurrence, Cohort, Fast track (FDA), ATM, Clostridioides difficile infection, Allo, GI, Therapy, Data, Patient, Infection, CDI, HSCT, ID, G&A, Pharmaceutical industry

Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today reported first quarter 2024 financial results and provided business updates.

Key Points: 
  • Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, today reported first quarter 2024 financial results and provided business updates.
  • These clinical results could further validate the vast potential of microbiome therapeutics in preventing adverse outcomes linked to gastrointestinal pathogens,” continued Mr. Shaff.
  • In the first quarter of 2024, approximately 1,411 completed prescription enrollment forms were received for VOWST and there were approximately 1,083 new patient starts.
  • Seres’ share of the VOWST net loss for the first quarter of 2024 was $7.1 million, which was included in the Company’s operating results within Collaboration (profit) loss sharing—related party.

SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical trials in haematological cancer patients

Retrieved on: 
måndag, april 22, 2024
Bone marrow, FQR, CRISPR, Nature Biotechnology, Safety, Education, Pharmacokinetics, Infection, Neutropenia, Survival, AMR, Febrile neutropenia, Gene editing, Partnership, Bloodstream infections, Development, BMBF, Escherichia coli, Research and development, Patient, Quinolone antibiotic, Synthetic biology, HSCT, Pharmaceutical industry

Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.

Key Points: 
  • Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.
  • Despite the significant advances in hematologic cancer therapy over the past decade, infectious complications, and antimicrobial resistance (AMR) continue to pose significant threats to patients and clinical outcomes1.
  • Currently, there are no approved therapies for the prevention of bloodstream infections (BSIs) in hematological cancer patients.
  • SNIPR Biome is developing SNIPR001 to address this urgent unmet need to combat infections in hematological cancer patients.

SNIPR Biome receives funding from CARB-X to support advancement of CRISPR-medicine SNIPR001 into clinical trials in haematological cancer patients

Retrieved on: 
måndag, april 22, 2024
Bone marrow, FQR, CRISPR, Nature Biotechnology, Safety, Education, Pharmacokinetics, Infection, Neutropenia, Survival, AMR, Febrile neutropenia, Gene editing, Partnership, Bloodstream infections, Development, BMBF, Escherichia coli, Research and development, Patient, Quinolone antibiotic, Synthetic biology, HSCT, Pharmaceutical industry

Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.

Key Points: 
  • Fluoroquinolone is recommended in the US for prophylaxis of bacterial infections and febrile neutropenia in hematological cancer patients at high risk of neutropenia.
  • Despite the significant advances in hematologic cancer therapy over the past decade, infectious complications, and antimicrobial resistance (AMR) continue to pose significant threats to patients and clinical outcomes1.
  • Currently, there are no approved therapies for the prevention of bloodstream infections (BSIs) in hematological cancer patients.
  • SNIPR Biome is developing SNIPR001 to address this urgent unmet need to combat infections in hematological cancer patients.

Tr1X Announces FDA Clearance of First Investigational New Drug Application for TRX103, an Allogeneic Regulatory T-Cell Therapy to Treat Autoimmune Diseases

Retrieved on: 
onsdag, april 10, 2024
FDA, Inflammation, Crohn's disease, City, Cell, Safety, Food, Haematopoietic system, Hematology, Allotransplantation, IND, Entrepreneurship, Patient, Graft-versus-host disease, Infection, Immune system, HSCT, Associate, SAN, Autoimmunity, Investigational New Drug, Disease

The company plans to initiate a Phase 1 study of TRX103, an investigational allogeneic off-the-shelf Tr1 Treg therapy, for this indication in the second quarter of 2024.

Key Points: 
  • The company plans to initiate a Phase 1 study of TRX103, an investigational allogeneic off-the-shelf Tr1 Treg therapy, for this indication in the second quarter of 2024.
  • Additionally, the company is on track to submit an IND for TRX103 for patients with refractory Crohn's disease in the third quarter of 2024.
  • This should enable Tr1X to develop further pipeline candidates that address even larger patient populations with equally unmet medical needs."
  • "Allogeneic stem cell transplantation is the only curative treatment for many advanced blood cancers and genetic and acquired diseases.

Seres Therapeutics Announces Completion of Patient Enrollment for SER-155 Phase 1B Cohort 2 Clinical Trial in Allogenic HSCT

Retrieved on: 
tisdag, april 9, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, Disease, Allo, Infection, Incidence, Patient, Immune tolerance, Gastrointestinal tract, GI, David Berry (inventor), Mortality, Bacteria, HSCT, Bloodstream infections, Graft-versus-host disease, Therapy, Chronic liver disease, Safety, Immunodeficiency, Data, Pharmaceutical industry

Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, announced today that enrollment is complete in the placebo-controlled Cohort 2 of its Phase 1b trial of SER-155 in patients who received Allogeneic Hematopoietic Stem Cell Transplantation (Allo HSCT).

Key Points: 
  • Seres Therapeutics, Inc. (Nasdaq: MCRB), a leading microbiome therapeutics company, announced today that enrollment is complete in the placebo-controlled Cohort 2 of its Phase 1b trial of SER-155 in patients who received Allogeneic Hematopoietic Stem Cell Transplantation (Allo HSCT).
  • Infections are a leading cause of mortality and morbidity in this immunocompromised patient population.
  • Our pending clinical results could validate the promise of microbiome therapeutics to prevent poor outcomes associated with pathogens in the GI tract.
  • Study Cohort 2, which includes 45 participants, incorporates a randomized, double-blinded placebo-controlled 1:1 design to further evaluate safety and engraftment, as well as clinical outcomes.

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in ha[...]

Retrieved on: 
tisdag, april 9, 2024
Eurostat, Diagnosis, Civil service commission, Graft-versus-host disease, Medicine, Cell, Bone marrow, Risk, Haematopoiesis, Patient, Committee, Knowledge, Regulation, HSCT, EMA, IRIS, European, Leukemia, COMP, Immune system, European Commission, Consultant, Safety, Haematopoietic system, Blood, Marketing, Umbilical cord, CATS, Pharmaceutical industry

Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Key Points: 


Orphan designation: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate Treatment in haematopoietic stem cell transplantation, 20/04/2020 Positive

Molecular Partners Reports Corporate Highlights From Q4 2023 and Key Financials for Full Year 2023

Retrieved on: 
torsdag, mars 14, 2024
CD33, GSK, Patient, Macrophage, Society, CD3, CD47, DLL3, Therapy, Molecular Partners, TME, Partner, DRS, ASH, AML, SNMMI, Partnership, RDT, Radionuclide, Tetra, Novartis, HSCT, MHS, FTE, Exposition, Delta, Safety, SITC, Amgen, Hematopoietic stem cell transplantation, FAP, Disease, Nuclear medicine, Immunotherapy, District court, Neoplasm, EANM, Alpha particle, JNJ, Tumor microenvironment, Gene family, CD70, AACR, CD40, CHF, CD123, NK, Engineering, Antigen, Molecule, Annual general meeting, Toxicity, CD16, B cell, Conference, MBA, DARPin, Data, Molecular imaging, Pharmaceutical industry

ZURICH-SCHLIEREN, Switzerland and CONCORD, Mass., March 14, 2024 (GLOBE NEWSWIRE) -- Ad hoc announcement pursuant to Art. 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced its corporate highlights and audited financial results for the full year 2023.

Key Points: 
  • 53 LR Molecular Partners AG (SIX: MOLN; NASDAQ: MOLN), a clinical-stage biotech company developing a new class of custom-built protein drugs known as DARPin therapeutics, today announced its corporate highlights and audited financial results for the full year 2023.
  • “2023 was a year of successful innovation and execution on our strategy, focusing on novel mechanisms that we believe only DARPin therapies can deliver.
  • In addition to the above updates, Molecular Partners continued to progress its RDT portfolio of projects in partnership with Novartis.
  • In the financial year 2023, Molecular Partners recognized total revenues and other income of CHF 7.0 million (2022: CHF 189.6 million) and incurred total expenses of CHF 68.1 million (2022: CHF 73.0 million).