Gaucher's disease

Freeline to Present New Data on its Gaucher and Parkinson’s Disease Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

Retrieved on: 
Wednesday, April 24, 2024
Bone pain, Congress, Gaucher's disease, Glucocerebrosidase, News, Abstract, Cell, Fracture, Patient, Gene, Society, Annual general meeting, Therapy, Pharmaceutical industry

LONDON, April 24, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics today announced the acceptance of multiple abstracts, including one for a late-breaking oral presentation on new clinical data from its Gaucher disease program, at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, Maryland.

Key Points: 
  • LONDON, April 24, 2024 (GLOBE NEWSWIRE) -- Freeline Therapeutics today announced the acceptance of multiple abstracts, including one for a late-breaking oral presentation on new clinical data from its Gaucher disease program, at the upcoming American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting taking place May 7-11, 2024 in Baltimore, Maryland.
  • “FLT201, our investigational gene therapy for Gaucher disease, has the potential to deliver better efficacy with a one-time treatment, and we are excited to present new clinical data from our ongoing Phase 1/2 trial in a late-breaking oral presentation at ASGCT.
  • We will also be sharing new data from our Parkinson’s disease research program, which builds on our work in Gaucher disease by leveraging the enhanced stability of our rationally engineered GCase enzyme.
  • The presentation materials will be available on the News & Events section of Freeline’s website following presentation at the ASGCT Annual Meeting.

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Retrieved on: 
Tuesday, April 9, 2024
Eurostat, Diagnosis, Civil service commission, Brain, B cell, Gaucher's disease, Cell, CD34, Bone marrow, Bleeding, Disease, Seizure, Bruise, Patient, Committee, Knowledge, Fracture, Spleen, Regulation, Fatigue, Autotransplantation, EMA, IRIS, Human, Life expectancy, Eye, Bone pain, Anemia, European, COMP, Sponsor, European Commission, Safety, PPD, Marketing, Liver, Medicine, Pharmaceutical industry

Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

Key Points: 


Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta Treatment of Gaucher disease, 21/08/2020 Positive

CENTOGENE Extends Strategic Partnership With Takeda to Continue Providing Access to Genetic Testing for Patients With Lysosomal Storage Disorders

Retrieved on: 
Tuesday, March 19, 2024
Diagnosis, Gaucher's disease, Disease, Partnership, Fabry, Shire (pharmaceutical company), LSD, Patient, Hunter syndrome, Glycogen storage disease, Fabry disease, Public Investment Fund, Neurodegenerative disease, Pharmaceutical industry

and ROSTOCK, Germany and BERLIN, March 19, 2024 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the extension of an ongoing partnership with Takeda (TSE: 4502/NYSE: TAK) to diagnose patients with Lysosomal Storage Disorders (LSDs).

Key Points: 
  • and ROSTOCK, Germany and BERLIN, March 19, 2024 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced the extension of an ongoing partnership with Takeda (TSE: 4502/NYSE: TAK) to diagnose patients with Lysosomal Storage Disorders (LSDs).
  • Under the renewed agreement, CENTOGENE will continue to provide Takeda with access to diagnostic testing for patients around the world.
  • The agreement was established to enhance patient access to rapid and reliable diagnostics for LSDs, including Fabry disease, Gaucher disease, and Hunter syndrome.
  • “Extending our longstanding partnership with Takeda highlights the continuing need to accelerate diagnoses for LSD patients globally and the ability of CENTOGENE to fulfill this vital service,” said Ian Rentsch, CENTOGENE Chief Commercial Officer and General Manager - Pharma.

Natalia Gomez-Ospina, MD, PhD is the Recipient of the 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award from the ACMG Foundation for Genetic and Genomic Medicine

Retrieved on: 
Wednesday, March 13, 2024
Research, Partner, Brain, B cell, FACMG, Gaucher's disease, Assistant, Pediatric Research, American Journal, Patient, Medical genetics, DSM-IV codes, Glycogen storage disease, System, Young Scientist Award, Gene therapy, Doctor of Philosophy, Stanford University, Protein, Genome, American College, Mucopolysaccharidosis, Genomics, Society, Hospital, Genetic, Cell, Young, Genetic distance, Hurler syndrome, Lucile Packard Children's Hospital

BETHESDA, Md., March 13, 2024 /PRNewswire/ -- Natalia Gomez-Ospina, MD, PhD is the recipient of the ACMG Foundation for Genetic and Genomic Medicine's 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award—the "Watson Award"—named for the American College of Medical Genetics and Genomics first and longstanding executive director, Michael S. Watson, MS, PhD, FACMG.

Key Points: 
  • BETHESDA, Md., March 13, 2024 /PRNewswire/ -- Natalia Gomez-Ospina, MD, PhD is the recipient of the ACMG Foundation for Genetic and Genomic Medicine's 2024 Dr. Michael S. Watson Genetic and Genomic Medicine Innovation Award—the "Watson Award"—named for the American College of Medical Genetics and Genomics first and longstanding executive director, Michael S. Watson, MS, PhD, FACMG.
  • "I am honored to receive this award in recognition of my dedication to advancing genetic and genomic medicine and my commitment to making a positive difference in the lives of patients with genetic diseases.
  • This award affirms the need for innovative approaches to treat such diseases and the immense potential that genome editing has in this regard.
  • "Dr. Gomez-Espina is a promising physician-scientist whose work is befitting the Dr. Michael Watson Genetic and Genomic Medicine Innovation Award, an award that honors an individual whose work has had a significant impact on genetic and genomic medicine.

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.”

Orphan designation: Ambroxol hydrochloride Treatment of Gaucher disease, 08/11/2023 Positive

Retrieved on: 
Sunday, February 4, 2024
CATS, COMP, IRIS, Consultant, Gaucher's disease, EMA, European Commission, Patient, Committee, Pharmaceutical industry

Key facts

Key Points: 
  • Key facts
    - Active substance
    - Ambroxol hydrochloride
    - Intended use
    - Treatment of Gaucher disease
    - Orphan designation status
    - Positive
    - EU designation number
    - EU/3/23/2849
    - Date of designation
    - Sponsor
    CATS Consultants GmbH
    Patients' organisations
    For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
    European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:
    EMA list of opinions on orphan medicinal product designation
    EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Human medicines European public assessment report (EPAR): Cerdelga, eliglustat, Date of authorisation: 19/01/2015, Revision: 17, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Spleen, Patient, Liver, Medical Subject Headings, Marketing, Disease, INN, Metabolism, Fatigue, Heartburn, Fracture, ATC, Gaucher's disease, Capsule, International, Enzyme replacement therapy, Bone pain, Language, Mouth, CYP2D6, European Medicines Agency, Indigestion, EMS, Select, Instant messaging, Bruise, Safety, Anatomy, Anemia, IIA, Medical device

Human medicines European public assessment report (EPAR): Cerdelga, eliglustat, Date of authorisation: 19/01/2015, Revision: 17, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Cerdelga, eliglustat, Date of authorisation: 19/01/2015, Revision: 17, Status: Authorised

Syncona to Acquire Freeline Therapeutics

Retrieved on: 
Wednesday, November 22, 2023
Slate, ADS, Committee, Acquisition, Gaucher's disease, Court, Proxy, SVB Securities, P.C, AAV, Part, Mass meeting, Scheme, Negotiation, Patient, Note, LON

LONDON, Nov. 22, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that it has entered into a definitive agreement with a newly established portfolio company of Syncona Ltd (LON: SYNC), a leading life science investor focused on creating, building and scaling global leaders in life science, to acquire Freeline in an all-cash transaction. Under the agreement, the newly established portfolio company will acquire all shares of Freeline not currently owned by Syncona for $6.50 per American Depositary Share (ADS). This price values Freeline’s entire issued share capital at approximately $28.3 million and represents a 51% premium over the closing price prior to the announcement of Syncona’s initial proposal on October 18. The acquisition is subject to the approval of Freeline’s minority shareholders and is expected to close in the first quarter of 2024.

Key Points: 
  • Under the agreement, the newly established portfolio company will acquire all shares of Freeline not currently owned by Syncona for $6.50 per American Depositary Share (ADS).
  • We are confident this negotiated transaction for Syncona to acquire Freeline and support it as a private entity is in the best interest of the company, as well as shareholders, employees and ultimately patients,” said Julia P. Gregory, an independent director and Chair of the Special Committee.
  • is acting as US legal counsel to Syncona; and Simmons & Simmons LLP is acting as UK legal counsel to Syncona.
  • Freeline entered into a secured convertible loan note in the aggregate amount of up to $15 million with Syncona (the “Note”).

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

Retrieved on: 
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.

Freeline to Present New Clinical Data for FLT201 in Gaucher Disease in Oral Presentation at Upcoming ESGCT 30th Annual Congress

Retrieved on: 
Monday, October 16, 2023
Congress, Cell, Glucocerebrosidase, Gene, Safety, CEST, Gaucher's disease, AAV, ESGCT, European Society of Gene and Cell Therapy, Patient, Pharmaceutical industry

The oral presentation will include data on safety, tolerability, GCase enzyme activity, substrate, and hemoglobin and platelet levels from the first two patients treated with FLT201.

Key Points: 
  • The oral presentation will include data on safety, tolerability, GCase enzyme activity, substrate, and hemoglobin and platelet levels from the first two patients treated with FLT201.
  • Details of the oral presentation are below:
    Date and Time: Wednesday, October 25 from 14:30-16:30 CEST (8:30-10:30 a.m.
  • ET)
    Additional details on the meeting can be found at the ESGCT 30th Annual Congress website .
  • The presentation materials will be available on the Investors section of Freeline’s website following presentation at the ESGCT Congress.