ESGCT

Orchard Therapeutics Announces First Patient Randomized in Registrational Trial of OTL-203 for MPS-I Hurler Syndrome

Retrieved on: 
Monday, February 5, 2024
Partnership, Mortality, Hurler, Orchard, Growth, Congress, Cell, Division, Heart, ERT, Glycosaminoglycan, GAG, Health, Disease, Patient, Safety, Transplant, FDA, EMA, Șaeș, San Raffaele Hospital, ESGCT, HSCT, Food, POC, Therapy, MPS, Hurler syndrome, Kyowa Kirin, Acquisition, Enzyme replacement therapy, European Medicines Agency, Gene, University, Eye, IDUA, HSC, RPD, Pharmaceutical industry

TOKYO, LONDON and BOSTON, Feb. 05, 2024 (GLOBE NEWSWIRE) -- Orchard Therapeutics, recently acquired by Kyowa Kirin with the goal of accelerating the delivery of new gene therapies to patients around the globe, today announced the first patient has been randomized at the M Health Fairview Masonic Children’s Hospital in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic stem cell (HSC) gene therapy, in patients with the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). The trial, referred to as HURCULES, compares treatment with OTL-203 to standard of care with allogeneic hematopoietic stem cell transplant (HSCT), and is expected to enroll 40 MPS-IH patients at sites across the U.S. and Europe.

Key Points: 
  • Approximately 60 percent of children born with MPS-I have the most severe subtype, MPS-IH, also called Hurler syndrome, and rarely live past the age of 10 when untreated.
  • Current treatment options for MPS-IH include allogeneic hematopoietic stem cell transplant (HSCT) and enzyme replacement therapy (ERT), both of which have significant limitations.
  • One patient experienced an acute hypersensitivity reaction that was considered probably related to HSC gene therapy.
  • The viral vector integration profile was comparable with other Orchard Therapeutics lentiviral-based HSC gene therapy studies, and all participants had a stable and highly polyclonal repertoire.

Forge Biologics Announces Positive FBX-101 Clinical Trial Update in Patients with Krabbe Disease Identified by Newborn Screening Ahead of RUSP Vote

Retrieved on: 
Monday, January 29, 2024
Health, Genetics, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Mortality, Death, HSCT, Patient, Legislation, GALC, Advisory Committee, AAV, Psychosine, ESGCT, Data, Gene, DSM-IV codes, Neurodegenerative disease, Disease, Safety, Central nervous system, Sphingolipid, Physician, Krabbe disease, Cell, Human services, Parent, Nervous system, Clinical trial, Forge, Peripheral nervous system, Hematopoietic stem cell transplantation, Brain, UCBT, Pharmaceutical industry

Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.

Key Points: 
  • Infantile Krabbe patients, often not diagnosed until after significant disease manifestations have occurred, typically die by the age of two if not treated by HSCT before symptoms are observed.
  • Previously published data have demonstrated that patients with Krabbe treated with HSCT demonstrate increased lifespan and stabilization of neurodegenerative disease in the central nervous system.
  • FBX-101, an investigational adeno-associated viral (AAV) gene therapy, has been designed to address the peripheral nerve disease not corrected by HSCT.
  • As a result, after a year of patient and foundation advocacy, Krabbe disease is again being voted on for potential inclusion on the RUSP.

Orchard Therapeutics Receives U.S. FDA Fast Track Designation for OTL-203 in MPS-IH

Retrieved on: 
Thursday, November 30, 2023
Quality of life, Glycosaminoglycan, Food, ESGCT, Health, Antibody, FDA, Cell, Disease, Mortality, Tissue, Congress, Gene, HSC, POC, Therapy, RPD, Heart, Hurler syndrome, Eye, San Raffaele Hospital, Death, San Raffaele, ERT, European Medicines Agency, Cognitive development, IDUA, Growth, HSCT, Patient, Safety, Diagnosis, Hurler, Fast Track, Pharmaceutical industry

BOSTON and LONDON, Nov. 30, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to OTL-203, an investigational hematopoietic stem cell gene (HSC) therapy being developed for the potential treatment of the Hurler subtype of mucopolysaccharidosis type I (MPS-IH). Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs. Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.

Key Points: 
  • Fast Track designation is intended to expedite the development of promising medicines that address serious medical needs.
  • Therapeutic candidates that receive Fast Track designation may be eligible for enhanced interactions with the FDA, including potentially quicker submission and review timelines.
  • “We are encouraged that OTL-203 has been granted Fast Track designation because new treatment options are urgently needed for children with MPS-IH, as the current standard of care is associated with significant morbidity and mortality.
  • Throughout the PoC study, treatment with OTL-203 has been generally well-tolerated with a safety profile consistent with the selected conditioning regimen.

Taysha Gene Therapies Reports Third Quarter 2023 Financial Results and Provides Corporate and Clinical Updates

Retrieved on: 
Tuesday, November 14, 2023
CNS, Webcast, Principal, ID, Associate, Gait, Safety, Pediatrics, Gene, Silicon Valley Bank, Socialization, Phenotype, Disease, Risk, Clinical trial, CHU, Administration, Patient, Cell, Dysrhythmia, FAAP, Research, ESGCT, Movement, Posture, Seizure, Hyperventilation, Development, IDMC, FRCP, Rett syndrome, Congress, Central nervous system, Mutation, Walking, Șaeș, AAV, MECP2, Pharmaceutical industry, Nursing, Vaccine, Cryptocurrency, Rett, DALLAS, Neuroscience

Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills.

Key Points: 
  • Following treatment, both patients experienced improvement in key clinical domains impacting activities of daily living, including breathing dysrhythmia, autonomic function, socialization, and gross and fine motor skills.
  • Additional information on available clinical data is available in the Company’s quarterly report on Form 10-Q for the quarter ended September 30, 2023, to be filed with the SEC.
  • Cash and cash equivalents: As of September 30, 2023, the Company had cash and cash equivalents of $164.3 million.
  • Taysha management will hold a conference call and webcast today at 4:30 pm ET to review its financial and operating results and to provide corporate and clinical updates.

MeiraGTx Announces Third Quarter 2023 Financial and Operational Results

Retrieved on: 
Tuesday, November 14, 2023
CNS, DSM-IV codes, DNA, Conditional sentence, Janssen Pharmaceuticals, Subthalamic nucleus, Patient, Research, ESGCT, FTD, Capsid, Quality control, Congress, I&I, Safety, Treatment, IND, Immunology, Inherited disorders of trafficking, ROFN, Evercore, Genome, STN, Central nervous system, Martin Lipton, Frontotemporal dementia, Xerostomia, Johnson & Johnson, MIA, GMP, Gene, BLA, Commercial, Insurance, Parotid gland, ALS, Parkinson's disease, Clinical trial, Obesity, QC, File, CGMP, RIX, Pharmaceutical industry, Vaccine, Cryptocurrency, Birth control, Sanofi, Janssen, Inflammation

Bota-vec for the Treatment of XLRP:

Key Points: 
  • Bota-vec for the Treatment of XLRP:
    Enrollment completed in second quarter 2023 in the pivotal Phase 3 LUMEOS clinical trial in collaboration with Janssen Pharmaceuticals, Inc. (Janssen), a Johnson & Johnson company.
  • In September 2023, the IND for bota-vec for the treatment of XLRP was transferred from MeiraGTx to Janssen.
  • Results from AQUAx Phase 1 open-label, dose-escalation study of gene therapy with AAV2-hAQP1 as a treatment for RIX and parotid gland hypofunction presented at ESGCT 2023 Annual Congress on October 26, 2023.
  • License revenue was $5.1 million for the quarter ended September 30, 2023, compared to $4.8 million for the quarter ended September 30, 2022.

Orchard Therapeutics Reports Third Quarter 2023 Financial Results and Highlights Recent Business Accomplishments

Retrieved on: 
Monday, November 13, 2023
Infant, Acquisition, HSC, Investment, Patient, Cell, ADS, ESGCT, U.S. FDA, FTD, Marketing, ICER, Diagnosis, R, Congress, Priority review, CVR, Food, Public, MLD, Abstract, Public health, Biomarker, MPS, Therapy, BLA, Gene, Health, HAE, Frontotemporal dementia, Hurler, Research, Disease, FDA, American depositary receipt, File, Hereditary angioedema, CTA, PDUFA, Society, Kyowa Kirin, Pharmaceutical industry, Security (finance), Fine chemical, Mobile phone, Vaccine, Dietary supplement, Cryptocurrency, Metabolism, Orchard, Atidarsagene autotemcel

Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024

Key Points: 
  • Biologics License Application for OTL-200 in MLD accepted by U.S. FDA under Priority Review; PDUFA date set for March 18, 2024
    BOSTON and LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced several business accomplishments along with its financial results for the quarter ended September 30, 2023.
  • Following the completion of the acquisition, Orchard Therapeutics will become a wholly-owned subsidiary of Kyowa Kirin.
  • The company had approximately 227.3 million ordinary shares, equivalent to 22.7 million American Depositary Shares, outstanding as of September 30, 2023.
  • As a result of the anticipated acquisition by Kyowa Kirin, Orchard Therapeutics has removed its financial guidance around Libmeldy revenue and anticipated cash runway.

Achilles Therapeutics Reports Third Quarter 2023 Financial Results and Recent Highlights

Retrieved on: 
Monday, November 13, 2023
ACHL, Annual general meeting, Therapy, Gene, Patient, Cell, Research, ESGCT, G&A, Immunotherapy, CD4, Blood, Conference, CD8, Cancer, Vaccine, SITC, R, Congress, Society, TIL, NSCLC, Pharmaceutical industry, Cryptocurrency, CNET

LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens to treat solid tumors, today announced its financial results for the quarter ended September 30, 2023, and recent business highlights.

Key Points: 
  • LONDON, Nov. 13, 2023 (GLOBE NEWSWIRE) -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing AI-powered precision T cell therapies targeting clonal neoantigens to treat solid tumors, today announced its financial results for the quarter ended September 30, 2023, and recent business highlights.
  • Research and development (R&D) expenses: R&D expenses were $14.7 million for the third quarter ended September 30, 2023, an increase of $4.1 million compared to $10.6 million for the third quarter ended September 30, 2022.
  • General and administrative (G&A) expenses: G&A expenses were $4.4 million for the third quarter ended September 30, 2023, a decrease of $1.0 million compared to $5.4 million for the third quarter ended September 30, 2022.
  • Net loss: Net loss for the third quarter ended September 30, 2023, was $16.7 million or $0.42 per share compared to $12.5 million or $0.32 per share for the third quarter ended September 30, 2022.

Generation Bio Reports Business Highlights and Third Quarter 2023 Financial Results

Retrieved on: 
Thursday, November 9, 2023
Cytokine, DNA, Spleen, D, Haemophilia, Gene, Therapeutic index, Liver, Tissue, Mouse, Cell, Research, ESGCT, G&A, Messenger, LNP, Innate lymphoid cell, NHP, Moderna, R, Congress, RNA, Vaccine

CAMBRIDGE, Mass., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Generation Bio Co. (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and third quarter 2023 financial results.

Key Points: 
  • (Nasdaq: GBIO), a biotechnology company innovating genetic medicines for people living with rare and prevalent diseases, reported business highlights and third quarter 2023 financial results.
  • “The recent advances in our immune-quiet DNA (iqDNA) and cell-targeted lipid nanoparticle (ctLNP) platforms represent two breakthroughs in the field of genetic medicine,” said Geoff McDonough, chief executive officer of Generation Bio.
  • R&D Expenses: Research and development (R&D) expenses were $21.9 million for the quarter ended September 30, 2023, compared to $21.2 million for the quarter ended September 30, 2022.
  • G&A Expenses: General and administrative (G&A) expenses were $11.6 million for the quarter ended September 30, 2023, compared to $11.5 million for the quarter ended September 30, 2022.

Precision BioSciences Announces Approval of First Clinical Trial Application of ARCUS Gene Insertion Program by Partner iECURE

Retrieved on: 
Wednesday, December 13, 2023
Research, Infectious Diseases, Genetics, Clinical Trials, Biotechnology, Health, Pharmaceutical, General Health, Science, Precision BioSciences, Nerve, Gene editing, Ornithine transcarbamylase deficiency, HDR, Gene, Clinical trial, Coma, IND, Liver, Disease, Risk, Patient, Cell, ESGCT, Blood, Hepatocyte, Primate, CRISPR, Ammonia, Death, Neurology, Congress, CTA, OTC, Precision, Metabolic disorder, Vaccine

“The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.

Key Points: 
  • “The acceptance of iECURE’s Clinical Trial Application marks an important milestone for patients with OTC deficiency and for both the iECURE and Precision BioSciences teams.
  • This is the first ARCUS in vivo gene editing program to progress into the clinic,” said Michael Amoroso, Chief Executive Officer of Precision Biosciences.
  • A 5% threshold of enzymatic activity has the potential for clinical benefit as previously identified in Annals of Clinical and Translational Neurology .
  • “ECUR-506 is the first of several partnered and wholly owned gene editing programs leveraging ARCUS in differentiated indications and we look forward to initial clinical data from this program.”

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

Retrieved on: 
Wednesday, November 8, 2023
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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.