European Society for Primary Immunodeficiencies

Beam Therapeutics Reports Pipeline and Business Updates and Third Quarter 2023 Financial Results

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Wednesday, November 8, 2023
Stem cell, FDA, Eli Lilly and Company, IND, Food, SCD, Trial of the century, Hematology, Biotechnology, LNP Media Group, Congress, Business development, G&A, Health, Thalassemia, Liver, Research and development, Sickle cell disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Research, CD34, Piz, R, Nature, Growth, Cell, Clinical trial, Gene, Partnership, LNP, PCSK9, ESGCT, AAT, Transplant, General Administration of Sport of China, AATD, Stock, GMP, Safety, European Society for Primary Immunodeficiencies, Investment, ANGPTL3, Patient, Drug discovery, Toxicity, Potential future exposure, ESCAPE, Administration, Cardiovascular disease, Pharmaceutical industry, Medical imaging, Vaccine, Lilly, Beam, Verve

CAMBRIDGE, Mass., Nov. 08, 2023 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported third quarter 2023 financial results and provided an update on the business and its clinical and pipeline progress.

Key Points: 
  • Beam anticipates that currently consented patients are sufficient to both fill the sentinel cohort (n=3) and to initiate the expansion cohort.
  • Research & Development (R&D) Expenses: R&D expenses were $100.0 million for the third quarter of 2023, compared to $85.3 million for the third quarter of 2022.
  • General & Administrative (G&A) Expenses: G&A expenses were $25.4 million for the third quarter of 2023, compared to $21.8 million for the third quarter of 2022.
  • Net Loss: Net loss was $96.1 million for the third quarter of 2023, or $1.22 per share, compared to $109.6 million for the third quarter of 2022, or $1.56 per share.

AlloVir Reports Third Quarter 2023 Financial Results

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Thursday, November 2, 2023
Science, Other Science, Biotechnology, Research, Pharmaceutical, Health, Infectious Diseases, Clinical Trials, RSV, Immunodeficiency, Trial of the century, Respiratory syncytial virus, Viral, Congress, Data, BKV, European Society for Primary Immunodeficiencies, Investment, Patient, Organ transplantation, Pharmaceutical industry, Cryptocurrency

AlloVir, Inc. (Nasdaq: ALVR), a late-clinical stage allogeneic T cell immunotherapy company, today reported financial results from the third quarter ended September 30, 2023.

Key Points: 
  • AlloVir, Inc. (Nasdaq: ALVR), a late-clinical stage allogeneic T cell immunotherapy company, today reported financial results from the third quarter ended September 30, 2023.
  • “At AlloVir, we are dedicated to serving immunocompromised patients suffering from devastating and life-threatening viral diseases,” said Diana Brainard, M.D., Chief Executive Officer, AlloVir.
  • Research and development expenses were $34.2 million for the quarter ended September 30, 2023, compared with $30.0 million for the quarter ended September 30, 2022.
  • General and administrative expenses were consistent at $12.8 million for the quarter ended September 30, 2023, compared with $12.9 million for the quarter ended September 30, 2022.

Sangamo Therapeutics Announces Strategic Update and Reports Third Quarter 2023 Financial Results

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Wednesday, November 1, 2023
Health, General Health, Research, Pharmaceutical, Science, Biotechnology, Regenerative Medicine Advanced Therapy, Ethics, Therapy, Pfizer, Gene, Partnership, Head, Exposition, European Society of Gene and Cell Therapy, ALTA, AAV, Haemophilia, Hope, 65th Street Yard, Workforce, ERT, DSM-IV codes, Cell, CAR, U.S. FDA, Safety, Investment, Patient, Central nervous system, Nav1.7, MAA, 22q13 deletion syndrome, Conference, Enzyme replacement therapy, Hematology, Quality of life, Mace Head Atmospheric Research Station, Novartis, Brain, European Society for Primary Immunodeficiencies, Haemophilia A, Spectrum, FDA, IND, Fabry disease, Prion, Society, Senior, SCN2A, ESGCT, STAAR, Intellectual disability, Sclerosis, BLA, Pharmaceutical industry, Management, Vaccine, Biogen, Neuroscience, Fabry, Sangamo Therapeutics

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.

Key Points: 
  • Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced recent business highlights, including progress on its strategic transformation and a corresponding restructuring of operations and workforce reduction, and reported third quarter 2023 financial results.
  • As a result of this restructuring, Sangamo is reducing its US workforce by approximately 40%, or approximately 162 roles.
  • Sangamo expects to incur approximately $8 million-$10 million in one-time restructuring costs in the fourth quarter of 2023.
  • Revenues for the third quarter ended September 30, 2023 were $9.4 million, compared to $26.5 million for the same period in 2022.

Orchard Therapeutics Announces Presentation of Additional Positive Data from Proof-of-concept Study of OTL-203 in MPS-IH at ESGCT 2023

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Thursday, October 26, 2023
Glycosaminoglycan, Heart, Hurler syndrome, Hearing loss, Congress, Photophobia, POC, ERT, IDUA, Cell, Disease, MPS, Antibody, Gene, San Raffaele Hospital, HSC, The New England Journal of Medicine, ESGCT, Eye, Safety, European Society for Primary Immunodeficiencies, Therapy, Patient, Diagnosis, Death, Hurler, San Raffaele, European Society of Gene and Cell Therapy, HSCT, Pharmaceutical industry, Medical device, Medical imaging

The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.

Key Points: 
  • The data were presented at the European Society of Gene and Cell Therapy (ESGCT) 30th Annual Congress taking place October 24-27, 2023, in Brussels.
  • “The complications associated with MPS-IH involve multiple organ systems and have an adverse impact on patients’ quality of life.
  • Importantly, following treatment with OTL-203, no patients reported photophobia (light sensitivity), or any other ophthalmological symptoms typically associated with MPS-IH.
  • Secondary endpoints include biochemical markers, additional clinical assessments, as well as safety and tolerability.

Ascendis Presents Updated and New TransCon™ IL-2 β⁄γ Monotherapy and Combination Therapy Data Confirming Clinical Activity Across Tumor Types at ESMO 2023

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Thursday, October 26, 2023
Patient, SCLC, Cmax, ESMO, Pembrolizumab, Colorectal cancer, European Society for Medical Oncology, Eosinophil, Carcinoma, Northern Transcon, Clinical, Oncology, Safety, European Society for Primary Immunodeficiencies, Interleukin, PR, Pharmaceutical industry, Extractive metallurgy

COPENHAGEN, Denmark, Oct. 26, 2023 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) reported updated and new data from its ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 β⁄γ in a poster presentation at ESMO 2023, the annual meeting of the European Society of Medical Oncology held in Madrid, Spain. The data included longer-term follow up of previously presented TransCon IL-2 β⁄γ monotherapy data from the IL-Believe Trial, and was the first presentation of dose escalation data informing recommended Phase 2 dose (RP2D) for TransCon IL-2 β⁄γ in combination with a checkpoint inhibitor.

Key Points: 
  • The data included longer-term follow up of previously presented TransCon IL-2 β⁄γ monotherapy data from the IL-Believe Trial, and was the first presentation of dose escalation data informing recommended Phase 2 dose (RP2D) for TransCon IL-2 β⁄γ in combination with a checkpoint inhibitor.
  • As of the August 15, 2023 data cutoff, 46 patients were enrolled into dose escalation: 25 to monotherapy and 21 to combination therapy.
  • RP2D for IL-Believe is 120 µg/kg of TransCon IL-2 β⁄γ administered intravenously every three weeks in both the monotherapy and combination therapy arms.
  • Additional details and highlights from the ESMO poster are available on the Investor & News section of the Ascendis Pharma website at https://investors.ascendispharma.com.

Freeline Presents Positive New Data from Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease at ESGCT 30th Annual Congress

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Wednesday, October 25, 2023
Bone pain, Research, Plasma, Clinical trial, AAV, DBS, Biomarker, AST, Congress, Liver, Cancer Treatment Centers of America, CEST, Cell, Enzyme replacement therapy, White blood cell, Tissue, Gene, Glucocerebrosidase, Blood, ESGCT, Gaucher's disease, Safety, European Society for Primary Immunodeficiencies, Lysosome, ALT, Pharmaceutical industry, Patient

LONDON, Oct. 25, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that new clinical data from its ongoing Phase 1/2 GALILEO-1 trial of FLT201, its adeno-associated virus (AAV) gene therapy candidate for Gaucher disease, show a substantial reduction of glucosylsphingosine (lyso-Gb1) levels in the blood of the first patient treated with FLT201. Lyso-Gb1 is a well-established biomarker of clinical response in Gaucher disease, with reductions in lyso-Gb1 correlating with positive clinical outcomes. These data are being highlighted in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress held in Brussels, Belgium.

Key Points: 
  • Lyso-Gb1 is a well-established biomarker of clinical response in Gaucher disease, with reductions in lyso-Gb1 correlating with positive clinical outcomes.
  • These data are being highlighted in an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) 30th Annual Congress held in Brussels, Belgium.
  • Reduction in lyso-Gb1 in the blood is strongly correlated with improvement in clinical outcomes in Gaucher disease.
  • The presentation entitled “Results from GALILEO-1, a First-In-Human Clinical Trial of FLT201 Gene Therapy in Patients with Gaucher Disease Type 1” is part of a session taking place from 14:30-16:30 CEST (8:30-10:30 a.m.

Janssen to Highlight Latest Advances in Retina Portfolio at the European Society of Retina Specialists (EURETINA) 2023 Annual Meeting

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Tuesday, October 3, 2023
Abstract, Abstract (summary), AMD, Geographic atrophy, Quality of life, Janssen, Research, Paratriathlon, GA, Patient, Peripheral vision, Johnson & Johnson, European Society for Primary Immunodeficiencies, Janssen Pharmaceuticals, Pharmaceutical industry, Retina

RARITAN, N.J., Oct. 3, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the European Society of Retina Specialists (EURETINA) 2023 annual meeting, taking place in Amsterdam, from October 5-8. The Company's presentations will include safety analysis data from two Phase 1 trials of the investigational gene therapy JNJ-81201887 (JNJ-1887): one trial in patients with geographic atrophy (GA), an advanced stage and severe form of age-related macular degeneration (AMD), and one trial in wet AMD (Abstracts #CA231214 and #CA231118).1,2 In addition, new research highlighting descriptive analyses of real-world GA trial populations, patients' perspectives on the symptoms and impact of X-linked retinitis pigmentosa (XLRP) and comorbidities among inherited retinal dystrophy patients will also be shared (Abstracts #CA231637, #CA231346 and #CA23429).3,4,5

Key Points: 
  • Five abstracts to be presented, including new real-world research and data on Janssen's investigational gene therapy JNJ-1887
    RARITAN, N.J., Oct. 3, 2023 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that five company-sponsored presentations will be featured during the European Society of Retina Specialists (EURETINA) 2023 annual meeting, taking place in Amsterdam, from October 5-8.
  • The Company's presentations will include safety analysis data from two Phase 1 trials of the investigational gene therapy JNJ-81201887 (JNJ-1887): one trial in patients with geographic atrophy (GA), an advanced stage and severe form of age-related macular degeneration (AMD), and one trial in wet AMD (Abstracts #CA231214 and #CA231118).1,2 In addition, new research highlighting descriptive analyses of real-world GA trial populations, patients' perspectives on the symptoms and impact of X-linked retinitis pigmentosa (XLRP) and comorbidities among inherited retinal dystrophy patients will also be shared (Abstracts #CA231637, #CA231346 and #CA23429).3,4,5
    Experience the full interactive Multichannel News Release here:
    "These data at EURETINA 2023 help bring us one step closer to a new era of innovation in retina," said James List, M.D., Ph.D., Global Therapeutic Area Head, whose team oversees a portfolio of programs including Retina at Janssen Research & Development, LLC.
  • "We're committed to building on this momentum – and doing so with boldness and urgency – as we advance our mission to restore and preserve vision for those living with retinal diseases."
  • Abstracts can also be found on the EURETINA website.

New drug class prevents key ageing mechanism in organ transplants

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Sunday, September 17, 2023
Mayo, Human, Brigham and Women's Hospital, Research, Organ transplantation, Harvard Medical School, Transplant, Congress, Lymph, Deficit spending, Mouse, Physical fitness, Dasatinib, Hospital, Clinical professor, European Society for Primary Immunodeficiencies, Vaccine, Dentistry, Quercetin, Senolytic

ATHENS, Greece, Sept. 18, 2023 /PRNewswire/ -- A novel study has shown that Senolytics, a new class of drugs, have the potential to prevent the transfer of senescence, a key mechanism of ageing, in recipients of older donor organs.

Key Points: 
  • ATHENS, Greece, Sept. 18, 2023 /PRNewswire/ -- A novel study has shown that Senolytics, a new class of drugs, have the potential to prevent the transfer of senescence, a key mechanism of ageing, in recipients of older donor organs.
  • The pioneering research, presented today at the European Society for Organ Transplantation (ESOT) Congress 2023, opens promising avenues for expanding the organ donor pool and enhancing patient outcomes.
  • By transplanting older donor organs into younger recipients, researchers from Harvard Medical School and the Mayo Clinic investigated the role of transplantation in inducing senescence, a biological mechanism linked to ageing and age-related diseases.
  • The researchers conducted age-disparate heart transplants from both young (3 months) and old (18–21 months) mice into younger recipients.

New drug class prevents key ageing mechanism in organ transplants

Retrieved on: 
Sunday, September 17, 2023
Mayo, Human, Brigham and Women's Hospital, Research, Organ transplantation, Harvard Medical School, Transplant, Congress, Lymph, Deficit spending, Mouse, Physical fitness, Dasatinib, Hospital, Clinical professor, European Society for Primary Immunodeficiencies, Vaccine, Dentistry, Quercetin, Senolytic

ATHENS, Greece, Sept. 18, 2023 /PRNewswire/ -- A novel study has shown that Senolytics, a new class of drugs, have the potential to prevent the transfer of senescence, a key mechanism of ageing, in recipients of older donor organs.

Key Points: 
  • ATHENS, Greece, Sept. 18, 2023 /PRNewswire/ -- A novel study has shown that Senolytics, a new class of drugs, have the potential to prevent the transfer of senescence, a key mechanism of ageing, in recipients of older donor organs.
  • The pioneering research, presented today at the European Society for Organ Transplantation (ESOT) Congress 2023, opens promising avenues for expanding the organ donor pool and enhancing patient outcomes.
  • By transplanting older donor organs into younger recipients, researchers from Harvard Medical School and the Mayo Clinic investigated the role of transplantation in inducing senescence, a biological mechanism linked to ageing and age-related diseases.
  • The researchers conducted age-disparate heart transplants from both young (3 months) and old (18–21 months) mice into younger recipients.

CareDx Advances Global Transplant Patient Care at ESOT Congress 2023

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Thursday, September 14, 2023
Biotechnology, Surgery, Hospitals, Health, Clinical Trials, Abstract, DNA, Paris Institute for Advanced Study, Comparison, Nucleic acid thermodynamics, Transplant, Doctor of Philosophy, Necker–Enfants Malades Hospital, Organ transplantation, Teaching hospital, University Hospital of Zürich, Associate, Congress, Plasma, Lung transplantation, Nephrology, Local, MD, Patient, Risk, Medical University of Vienna, UZ Leuven, Harbin Medical University, Paris, European Society for Primary Immunodeficiencies, Caregiver, Dentistry, Medical device, Epidemiology

CareDx, Inc. (Nasdaq: CDNA) – The Transplant Company™ focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers – today announced a leading presence at the 2023 European Society for Organ Transplantation (ESOT) Congress taking place September 17-20, 2023, in Athens, Greece.

Key Points: 
  • CareDx, Inc. (Nasdaq: CDNA) – The Transplant Company™ focused on the discovery, development, and commercialization of clinically differentiated, high-value healthcare solutions for transplant patients and caregivers – today announced a leading presence at the 2023 European Society for Organ Transplantation (ESOT) Congress taking place September 17-20, 2023, in Athens, Greece.
  • CareDx is hosting an educational symposium for medical professionals demonstrating the clinical utility of AlloSeq cfDNA.
  • “We are proud to attend ESOT Congress 2023, a preeminent European transplant conference, and share the latest data demonstrating the use of AlloSeq cfDNA,” said Reg Seeto, CEO and President of CareDx.
  • CareDx will review the latest data on AlloSeq cfDNA during an ESOT Congress 2023 Industry Satellite Symposium titled, “Adopting dd-cfDNA for Solid Organ Surveillance: Raising the Standards of Transplant Care.” The symposium will be held live and streamed virtually on ESOT’s Congress platform on Sunday, September 17, 2023.