Gaucher's disease

Freeline Shareholders Approve Acquisition by Syncona

Retrieved on: 
Monday, February 12, 2024

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

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

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

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

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

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.

Global Enzyme Replacement Therapy (ERT) Market Research Report 2023-2032 Featuring Key Players - Biomarin Pharmaceutical, Leadiant Biosciences, Pfizer, Sanofi, & AbbVie - ResearchAndMarkets.com

Retrieved on: 
Thursday, October 5, 2023

The "Enzyme Replacement Therapy Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Enzyme Replacement Therapy Global Market Report 2023" report has been added to ResearchAndMarkets.com's offering.
  • In the forefront of medical advancement, the enzyme replacement therapy (ERT) market stands tall, offering vital treatment avenues for individuals grappling with chronic disorders arising from enzyme deficiencies.
  • The research report delves deep into the nuances of the ERT sector, highlighting market statistics, regional shares, and key players commanding significant market share.
  • The report also touches upon the market revenues, detailing contributions from key treatments like Sebelipase alfa, Elosulfase alfa, and more.

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

“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.

Freeline Reports Positive Initial Clinical Data from First Cohort of Phase 1/2 GALILEO-1 Trial of FLT201, Its Novel Gene Therapy Candidate, in Gaucher Disease

Retrieved on: 
Wednesday, October 4, 2023

LONDON, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported positive initial safety, tolerability and enzyme activity data from the ongoing Phase 1/2 GALILEO-1 trial evaluating FLT201, its adeno-associated virus (AAV) gene therapy candidate, in Gaucher disease. Gaucher disease is a debilitating genetic disorder in which a deficiency of the GCase enzyme leads to a buildup of harmful substrates, causing symptoms including enlarged spleen and liver, low blood counts, bone pain and reduced lung function. In addition to demonstrating a favorable safety and tolerability profile, data from the first two patients in GALILEO-1 show that a single infusion of FLT201 led to several hundred-fold increases in GCase activity in plasma and normalization of GCase activity in leukocytes.

Key Points: 
  • ET
    LONDON, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported positive initial safety, tolerability and enzyme activity data from the ongoing Phase 1/2 GALILEO-1 trial evaluating FLT201, its adeno-associated virus (AAV) gene therapy candidate, in Gaucher disease.
  • "These initial clinical data are very compelling,” said Pilar Giraldo, M.D., Ph.D., hematologist at the Spanish Foundation for the Study and Therapy of Gaucher Disease, Quirónsalud Hospital – Zaragoza, and an investigator in the GALILEO-1 trial.
  • “While existing therapies have had a significant impact on the disease, many patients continue to experience symptoms and current therapies come with a heavy lifelong treatment burden.
  • ET today to discuss these initial clinical data for FLT201 in Gaucher disease.

CENTOGENE Reports First Half 2023 Financial Results

Retrieved on: 
Thursday, September 7, 2023

and ROSTOCK, Germany and BERLIN, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its unaudited financial results for the six months ended June 30, 2023.

Key Points: 
  • and ROSTOCK, Germany and BERLIN, Sept. 07, 2023 (GLOBE NEWSWIRE) -- Centogene N.V. (Nasdaq: CNTG), the essential life science partner for data-driven answers in rare and neurodegenerative diseases, today announced its unaudited financial results for the six months ended June 30, 2023.
  • Cost of sales increased by €2,692 thousand, or 20.7%, to €15,728 thousand for the first half of 2023, from €13,036 thousand for the first half of 2022.
  • General administrative expenses decreased by €112 thousand, or 0.6%, to €17,172 thousand for the first half of 2023, from €17,284 thousand for the first half of 2022.
  • The decrease is mainly due to the increase in consumable expenses on approximately €1,601 thousand for the first half of 2023.