News – Page 2 – Bloomsbury Genetic Therapies

Bloomsbury Genetic Therapies Receives Rare Pediatric Disease Designation from the U.S. FDA for BGT-INAD for the Treatment of Infantile Neuroaxonal Dystrophy (INAD) and Announces Upcoming Type B Pre-IND Meeting with the U.S. FDA

29 August 2023

– RPDD and Type B pre-IND meeting granted by the FDA – London, UK, 29 August 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug […]

Bloomsbury Genetic Therapies Receives Rare Pediatric Disease Designation from the U.S. FDA for BGT-NPC for the Treatment of Niemann-Pick Disease Type C (NPC)

21 August 2023

– RPDD granted in the United States – London, UK, 21 August 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) has granted […]

Bloomsbury Genetic Therapies Receives Orphan Drug Designation from the U.S. FDA for BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

9 August 2023

London, UK, 9 August 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for BGT-OTCD, the Company’s investigational […]

Bloomsbury Genetic Therapies Receives Rare Pediatric Disease Designation from the U.S. FDA for BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

18 July 2023

– RPDD granted in the United States – London, UK, 18 July 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) has granted […]

Bloomsbury Genetic Therapies Completes Successful Scientific Advice Meeting with the MHRA regarding its BGT-INAD Program

26 June 2023

– Company intends to move forward with toxicology and biodistribution study in rodents ahead of a single, Phase 1/2/3 trial in Infantile Neuroaxonal Dystrophy (INAD) – London, UK, 26 June 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based […]

Bloomsbury Genetic Therapies Announces Publication of Article Highlighting Preclinical Data from BGT-NPC Program, Supporting BGT-NPC as a Potential Treatment for Niemann-Pick Disease Type C

14 June 2023

London, UK, 14 June 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that an article highlighting first preclinical data from BGT-NPC, as a potential new brain-targeted therapeutic for the treatment of […]

Bloomsbury Genetic Therapies Completes Successful Scientific Advice Meeting with the MHRA regarding its BGT-NPC Program

12 June 2023

– Company intends to move forward with toxicology and biodistribution study in rodents ahead of a single, Phase 1/2/3 trial in Niemann-Pick Disease Type C (NPC) – London, UK, 12 June 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases […]

Bloomsbury Genetic Therapies Outlines Pre-Clinical Data on CNS Programs Presented at the American Society of Gene and Cell Therapy 26th Annual Meeting

18 May 2023

London, UK, 18 May 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that pre-clinical data from two of its brain-targeted programs, BGT-INAD and BGT-NPC for the treatment of Infantile Neuroaxonal Dystrophy […]

Bloomsbury Genetic Therapies Announces Clinical Trial Application Accepted by U.K. Regulatory Agency to Initiate Phase 1/2 Gene Therapy Trial of BGT-OTCD for the Treatment of Ornithine Transcarbamylase Deficiency (OTCD)

15 May 2023

– First in-human phase 1/2 trial to evaluate the safety, efficacy and tolerability of BGT-OTCD in paediatric patients with confirmed diagnosis of OTCD – – University College London expects to initiate the clinical trial in Q3 2023 – London, UK, 15 May 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially […]

Bloomsbury Genetic Therapies Receives Rare Paediatric Disease Designation from the U.S. FDA for BGT-DTDS for the Treatment of Dopamine Transporter Deficiency Syndrome (DTDS)

3 May 2023

London, UK, 3 May 2023 – Bloomsbury Genetic Therapies Limited, a clinical-stage biotechnology company developing potentially curative treatments for patients suffering from rare neurological and metabolic diseases based on clinically proven gene therapy technologies, announced today that the U.S. Food and Drug Administration (FDA) has granted Rare Paediatric Disease (RPD) Designation for BGT-DTDS, the Company’s […]