Gaucher

PANTHERx®Rare Selected by Edenbridge Pharmaceuticals as Specialty Pharmacy for Yargesa® (miglustat 100 mg capsules)

Retrieved on: 
Monday, April 1, 2024
Disease, Capsule, Patient, Pharmacy, Bone pain, Hepatomegaly, Enzyme replacement therapy, Anemia, Gaucher, Thrombocytopenia, Liver, Spleen, Pharmaceutical industry

Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.

Key Points: 
  • Yargesa® is a glucosylceramide synthase inhibitor that exerts its effects through a process called substrate reduction therapy.
  • Through this process, Yargesa® reduces the buildup of harmful glycosphingolipids and symptoms of the disease.
  • This accumulation leads to manifestations including but not limited to enlarged spleen or liver, hepatomegaly, anemia, thrombocytopenia, and bone pain.
  • "We appreciate Edenbridge Pharmaceuticals' commitment to making a difference in the lives of patients with Type 1 Gaucher disease.

Freeline Shareholders Approve Acquisition by Syncona

Retrieved on: 
Monday, February 12, 2024
Acquisition, Court, Company register, Scheme, High court, Proxy, Therapy, Patient, Environment, Gaucher, Gaucher's disease, Mass meeting, AAV

LONDON, Feb. 12, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (“Freeline”) and Syncona Ltd (“Syncona”) today announced that Freeline’s shareholders have approved the proposal for Bidco 1354 Limited (“Bidco”), a wholly owned subsidiary of Syncona Portfolio Limited (“Syncona Portfolio”), to acquire all shares of Freeline not currently owned by Syncona Portfolio for $6.50 per American Depositary Share (“ADS”) in cash (the “Acquisition”).

Key Points: 
  • LONDON, Feb. 12, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (“Freeline”) and Syncona Ltd (“Syncona”) today announced that Freeline’s shareholders have approved the proposal for Bidco 1354 Limited (“Bidco”), a wholly owned subsidiary of Syncona Portfolio Limited (“Syncona Portfolio”), to acquire all shares of Freeline not currently owned by Syncona Portfolio for $6.50 per American Depositary Share (“ADS”) in cash (the “Acquisition”).
  • The resolution was passed by the requisite majority of Freeline shareholders and was accordingly passed.
  • Chris Hollowood, CEO of Syncona Investment Management Limited, said: “We are delighted our proposal to acquire Freeline has been approved by the required majority of Freeline shareholders.
  • We believe the acquisition by Syncona, which will result in Freeline becoming a private company, provides the best path forward for us to do that in this current environment, and we are grateful for the overwhelming support shown by our shareholders.”

Gain Therapeutics Announces Dosing of First Two Subjects in Phase 1 Clinical Trial of GT-02287, a Novel GCase-targeting Small Molecule Therapy for GBA1 Parkinson’s Disease

Retrieved on: 
Wednesday, October 4, 2023
Neuron, GBA1, Human, Index, Therapy, Pharmacokinetics, SAD, Institute of Genomics and Integrative Biology, Regenerative medicine, Gaucher, Dementia, Patient, Blood, Lewy body, MAD, MTD, Gaucher's disease, Glucocerebrosidase, Safety, Disease, Pharmaceutical industry, Vaccine

“Initiating first-in-human dosing with GT-02287 is an important milestone for Gain as we enter a new era as a clinical-stage company,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics.

Key Points: 
  • “Initiating first-in-human dosing with GT-02287 is an important milestone for Gain as we enter a new era as a clinical-stage company,” said Matthias Alder, Chief Executive Officer of Gain Therapeutics.
  • Based on these data, GT-02287 has the potential to slow or even stop the progression of Parkinson’s disease.
  • Gain’s broad pipeline of novel allosteric therapies, including GT-02287, was discovered via the Company’s SEE-Tx® drug discovery platform.
  • Designed to leverage AI-supported 3D structural biology and supercomputer-powered proprietary physics-based models, Gain is exploiting the untapped opportunities of allosteric binding sites and allosteric modulators to treat disease.

CENTOGENE Announces Publication Establishing Lyso-Gb1 as a Predictive Biomarker for Gaucher Disease Patients

Retrieved on: 
Friday, September 1, 2023
Gaucher's disease, GBA1, Diagnosis, Prognosis, GD, DBS, Patient, Collection, Physician, CNTG, Enzyme replacement therapy, Medical imaging, Gaucher

“Over the past decade, we have leveraged all our multiomic technological know-how to establish and confirm lyso-Gb1 as the best biomarker for Gaucher disease,” said Professor Peter Bauer, CENTOGENE’s Chief Medical and Genomic Officer.

Key Points: 
  • “Over the past decade, we have leveraged all our multiomic technological know-how to establish and confirm lyso-Gb1 as the best biomarker for Gaucher disease,” said Professor Peter Bauer, CENTOGENE’s Chief Medical and Genomic Officer.
  • “This landmark study further advances our understanding of Gaucher disease patients – demonstrating a significant correlation between lyso-Gb1 levels and disease severity.
  • The results of the study revealed a highly significant correlation between lyso-Gb1 and disease severity in all Gaucher patients, including those with novel rare GBA1 variants.
  • “The progressive increase in lyso-Gb1 levels in untreated Gaucher patients suggests that these patients could benefit from treatment, such as enzyme replacement therapy.

Freeline Reports Second Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, August 15, 2023
Gaucher's disease, GBA1, ADS, CMC, G&A, AAV, Sale, Patient, Administration, Research and development, R, Research, Glucocerebrosidase, Safety, Disease, Pharmaceutical industry, Gaucher, Daimler Freeline

LONDON, Aug. 15, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported financial results for the second quarter of 2023 and provided a business update.

Key Points: 
  • ET today
    LONDON, Aug. 15, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported financial results for the second quarter of 2023 and provided a business update.
  • Today, Freeline announced the dosing of the second patient in its GALILEO-1 trial of FLT201, marking the completion of dosing in the first cohort.
  • Cash Position: As of June 30, 2023, unrestricted cash and cash equivalents were $38.8 million, compared to $55.4 million as of March 31, 2023.
  • Freeline expects its current level of cash and cash equivalents will enable the company to fund its planned operations into the second quarter of 2024.

Freeline to Host Virtual KOL Event on its Gene Therapy Candidate, FLT201, in Gaucher Disease

Retrieved on: 
Tuesday, July 25, 2023
Gaucher's disease, KOL, Salford Royal Hospital, AAV, Outline, United Kingdom, Epidemiology, Safety, Pharmaceutical industry, Gaucher

LONDON, July 25, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced it will host a virtual Key Opinion Leader (KOL) event on FLT201, its adeno-associated virus (AAV) gene therapy candidate in Gaucher disease Type 1, on Tuesday, August 1, 2023 at 8:30 a.m.

Key Points: 
  • LONDON, July 25, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced it will host a virtual Key Opinion Leader (KOL) event on FLT201, its adeno-associated virus (AAV) gene therapy candidate in Gaucher disease Type 1, on Tuesday, August 1, 2023 at 8:30 a.m.
  • The event will feature Gaucher disease expert, Dr. Reena Sharma, Consultant Adult Metabolic Medicine and Honorary Senior Lecturer at the Salford Royal Hospital in the United Kingdom, who will discuss the epidemiology, treatment landscape, and unmet need in Gaucher disease.
  • The Freeline leadership team will provide a corporate overview and outline the clinical progress of FLT201.
  • A live webcast of the event will be available on the Investors section of Freeline’s website at www.freeline.life.

Freeline Announces First Patient Dosed with Its Novel Gene Therapy Candidate for Gaucher Disease and Unveils Research Program in GBA1-linked Parkinson’s Disease

Retrieved on: 
Monday, June 26, 2023
Tissue, Gaucher's disease, GBA1, Preclinical development, Lysosome, Clinical trial, Bone pain, AAV, Lung, Dementia, Patient, Residential treatment center, Risk, Liver, Research, Glucocerebrosidase, Safety, Disease, Vaccine, Pharmaceutical industry, Gaucher, Daimler Freeline

LONDON, June 26, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that the first patient has been dosed in the Phase 1/2 GALILEO-1 clinical trial evaluating FLT201, its adeno-associated virus (AAV) gene therapy candidate, in Gaucher disease type 1. Gaucher disease is a debilitating genetic disorder in which a deficiency of the GCase enzyme leads to a buildup of fatty substances in the organs, causing symptoms including enlarged spleen and liver, low blood counts, bone pain and reduced lung function.

Key Points: 
  • “We believe that FLT201 has life-changing potential for people with the most common type of Gaucher disease,” said Pamela Foulds, M.D., Chief Medical Officer at Freeline.
  • FLT201 is a highly differentiated gene therapy candidate for Gaucher disease type 1 that delivers a longer-acting, rationally engineered enzyme.
  • As in Gaucher disease, the GBA1 mutations lead to a deficiency of GCase and the accumulation of pathological substrates.
  • “There are no approved disease modifying therapies for Parkinson’s disease, and symptomatic treatments become less effective as the disease progresses.

AVROBIO Completes Sale of Cystinosis Gene Therapy Program for $87.5 Million

Retrieved on: 
Monday, June 12, 2023
Health, Biometrics, Stem Cells, Genetics, Other Health, Clinical Trials, Biotechnology, Wells Fargo, Gaucher, AVRO, HSC, Cystinosis, Glycogen storage disease type II, Hunter syndrome, TD, DSM-IV codes, Pharmaceutical industry, Novartis

(Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced the closing of the previously announced agreement to sell its investigational hematopoietic stem cell (HSC) gene therapy program for cystinosis to Novartis for $87.5 million in cash.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced the closing of the previously announced agreement to sell its investigational hematopoietic stem cell (HSC) gene therapy program for cystinosis to Novartis for $87.5 million in cash.
  • AVROBIO retains full rights to its portfolio of first-in-class HSC gene therapies for Gaucher disease type 1 and type 3, Hunter syndrome and Pompe disease.
  • Proceeds from this transaction are expected to extend the Company’s cash runway into the fourth quarter of 2024.
  • TD Cowen and Wells Fargo Securities, LLC acted as financial advisors to AVROBIO in the transaction.

Freeline Reports First Quarter 2023 Financial Results and Business Highlights

Retrieved on: 
Tuesday, May 30, 2023
Gaucher's disease, CMC, American depositary receipt, Sale, Parini, Daimler Freeline, Patient, Cell, Medicines and Healthcare products Regulatory Agency, Innovation, Safety, MHRA, Dedicated, Pharmaceutical industry, Gaucher, Brammer

LONDON, May 30, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported financial results for the first quarter of 2023 and provided a business update.

Key Points: 
  • LONDON, May 30, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported financial results for the first quarter of 2023 and provided a business update.
  • “The granting of the ILAP designation by UK regulators further highlights the significant need for more effective and less burdensome therapies for people with Gaucher disease.
  • “Importantly, we continue to make significant strides as a company in driving greater focus, financial discipline, and operational efficiency,” Parini continued.
  • Cash position: As of March 31, 2023, unrestricted cash and cash equivalents were $55.4 million, compared to $47.3 million as of December 31, 2022.

Freeline Granted ILAP Designation in United Kingdom by MHRA for FLT201, an Investigational Gene Therapy for the Treatment of Gaucher Disease

Retrieved on: 
Monday, May 22, 2023
Gaucher's disease, National Health Service, Health care, Healthcare Improvement Scotland, Wales, Marketing, MD, Patient, National Institute for Health and Care Excellence, Innovation, NHS, MAA, Therapy, NICE, SMC, MHRA, Disease, Pharmaceutical industry, Gaucher

Medicines and Healthcare products Regulatory Agency (MHRA).

Key Points: 
  • Medicines and Healthcare products Regulatory Agency (MHRA).
  • “We believe FLT201 has life-changing potential for people with Gaucher disease type 1, the most common type of the disease,” said Pamela Foulds, MD, Chief Medical Officer of Freeline.
  • Existing therapies come with a heavy life-long treatment burden, and even with treatment, many patients continue to experience serious symptoms.
  • We believe that FLT201 may improve outcomes for patients with a one-time gene therapy.