Gaucher's disease

Freeline Announces June 2022 Inducement Grant Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Friday, July 1, 2022
Research, GLOBE, Gaucher's disease, American, AAV, Company, Employment, Transgene, Nasdaq, Fabry disease

LONDON, July 01, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that onJune 30, 2022, the Company granted a newly hired employee non-statutory options to purchase 5,500 of the Companys ordinary shares.

Key Points: 
  • LONDON, July 01, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that onJune 30, 2022, the Company granted a newly hired employee non-statutory options to purchase 5,500 of the Companys ordinary shares.
  • The award was granted as an inducement material to the employee entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4) and was approved by an independent subcommittee of the remuneration committee of Freelines board of directors.
  • The award was granted pursuant to the terms of Freelines 2021 Equity Inducement Plan, which was adopted by Freelines board of directors inSeptember 2021.
  • Freeline is headquartered in the UK and has operations in Germany and the US.

Global Orphan Drugs Market Report to 2030 - Players Include Amgen, Bristol-Myers Squibb and Pfizer - ResearchAndMarkets.com

Retrieved on: 
Tuesday, June 14, 2022
Health, Pharmaceutical, Cystic fibrosis, Lists of diseases, Gaucher's disease, Multiple myeloma, Degenerative disease, AML, Pancreatic cancer, HAE, Hereditary angioedema, Ovarian cancer, DMD, Fabry disease, Growth, Hypoparathyroidism, Birth, Acute myeloid leukemia, Infection, Duchenne muscular dystrophy, Prevalence, Alzheimer's disease, Renal cell carcinoma, Hunter syndrome, Non-Hodgkin lymphoma, Haemophilia, Huntington's disease, CAGR, Pharmaceutical industry, Duchenne

The global orphan drugs market was valued at $1,40,000.0 million in 2020 and is projected to reach $4,35,686.3 million by 2030 registering a CAGR of 11.8% from 2021 to 2030.

Key Points: 
  • The global orphan drugs market was valued at $1,40,000.0 million in 2020 and is projected to reach $4,35,686.3 million by 2030 registering a CAGR of 11.8% from 2021 to 2030.
  • The pharmaceutical agents administered to treat, diagnose, and prevent life-threatening diseases (rare diseases) are known as orphan drugs.
  • In addition, favorable government policies, and availability of market exclusivity for orphan drugs developers further supplement the global orphan drugs market growth.
  • The global orphan drugs market is segmented on the basis of disease type, and region to provide a detailed assessment of the market.

Freeline Initiates Dosing of Second Cohort in B-LIEVE Dose Confirmation Trial of FLT180a in Hemophilia B

Retrieved on: 
Tuesday, June 14, 2022
Woman, ISTH, Conditional sentence, Thrombosis, Congress, Meta-analysis, Company, Bleeding, Research, Haemophilia, Haemophilia B, Man, Gaucher's disease, Transgene, Capsid, Forward-looking statement, World Federation of Hemophilia, International Society, Failure, Blood, Nasdaq, AAV, Physician, Fabry disease, GLOBE, Patient, Private Securities Litigation Reform Act, Infection, COVID-19, Factor IX, EDGAR, World government, MD, Prevalence, Form 6-K, X chromosome, U.S. Securities and Exchange Commission, International Society on Thrombosis and Haemostasis, FIX, Safety, Terminology, Vaccine, Pharmaceutical industry, Daimler Freeline

LONDON, June 14, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today announced that it has begun dosing the second cohort in its Phase 1/2 B-LIEVE dose confirmation trial of FLT180a in people with hemophilia B. Based on the strength of the data from cohort one and consistent with the advice of an Independent Data Monitoring Committee, patients in cohort two will receive the same low dose (7.7e11 vg/kg) of FLT180a and optimized prophylactic immune management regimen that were used in the first cohort. Dosing of the first cohort was completed in April and initial data from that cohort will be presented at the International Society on Thrombosis and Haemostasis (ISTH) Congress being held July 9-13, 2022 in London, UK.

Key Points: 
  • The data also provide confidence in the dosing for our pivotal Phase 3 trial, which we anticipate beginning in 2023 as planned.
  • FLT180a is on track to deliver proof of concept that our gene therapies have the potential to achieve our goal.
  • FLT180a has been studied in B-AMAZE, a Phase 1/2 dose-finding trial in patients with severe and moderately severe hemophilia B with the goal of normalizing FIX activity in patients with moderate and severe hemophilia B.
  • A Phase 1/2 dose-confirmation trial of FLT180a called B-LIEVE to finalize a dose for a Phase 3 pivotal trial is in progress.

Freeline Receives Nasdaq Deficiency Notice Regarding Minimum Bid Price Requirement

Retrieved on: 
Friday, June 3, 2022
Company, Research, Gaucher's disease, Transgene, Forward-looking statement, Notice, American, ADSS, AAV, U.S. Securities and Exchange Commission, Fabry disease, GLOBE, Private Securities Litigation Reform Act, Compliance, EDGAR, ADS, SEC, Form 6-K, Calendar, Terminology, Auction, Audit, Nasdaq

LONDON, June 03, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline) today disclosed the receipt of a notice (the Notice) on May 31, 2022 from the Nasdaq Stock Market LLC (Nasdaq) that the Company is not currently in compliance with the $1.00 minimum bid price requirement for continued listing of the Companys American Depositary Shares (the ADS) on the Nasdaq Global Select Market, as set forth in Nasdaq Listing Rule 5450(a)(1) (the Minimum Bid Price Requirement).

Key Points: 
  • LONDON, June 03, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline) today disclosed the receipt of a notice (the Notice) on May 31, 2022 from the Nasdaq Stock Market LLC (Nasdaq) that the Company is not currently in compliance with the $1.00 minimum bid price requirement for continued listing of the Companys American Depositary Shares (the ADS) on the Nasdaq Global Select Market, as set forth in Nasdaq Listing Rule 5450(a)(1) (the Minimum Bid Price Requirement).
  • The Notice indicated that, consistent with Nasdaq Listing Rule 5810(c)(3)(A), the Company has 180 days, or until November 28, 2022 (the Compliance Deadline), to regain compliance with the Minimum Bid Price Requirement by having the closing bid price of the Companys ADSs meet or exceed $1.00 per ADS for at least ten consecutive business days.
  • Freeline intends to monitor the closing bid price of its ADSs and may, if appropriate, consider implementing available options to regain compliance with the Minimum Bid Price Requirement.
  • All statements, other than historical facts, including statements regarding the Company's intention to resolve the deficiency and regain compliance with the Nasdaq listing rules, are forward-looking statements.

Freeline Announces May 2022 Inducement Grants Under Nasdaq Listing Rule 5635(c)(4)

Retrieved on: 
Friday, June 3, 2022
Company, Research, Gaucher's disease, Transgene, American, Nasdaq, AAV, Fabry disease, GLOBE, Employment

The awards were granted as an inducement material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4) and were approved by an independent subcommittee of the remuneration committee of Freelines board of directors.

Key Points: 
  • The awards were granted as an inducement material to the employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4) and were approved by an independent subcommittee of the remuneration committee of Freelines board of directors.
  • The awards were granted pursuant to the terms of Freelines 2021 Equity Inducement Plan, which was adopted by Freelines board of directors inSeptember 2021.
  • Freeline is a clinical-stage biotechnology company developing transformative adeno-associated virus (AAV) vector-mediated systemic gene therapies.
  • Freeline is headquartered in the UK and has operations in Germany and the US.

CytRx Announces Distribution of Series D Preferred Stock to Holders of Its Common Stock

Retrieved on: 
Thursday, May 19, 2022
Oncology, Health, Clinical Trials, Research, Science, Pharmaceutical, Biotechnology, Company, CytRx, Niemann–Pick disease, Securities and Exchange Commission (Nigeria), ImmunityBio, Annual report, Patient, Venture round, Nature, Delaware General Corporation Law, Research, FDA, Industry, EMA, Board, Forward-looking statement, Proxy, Board of directors, NPC, Therapy, Gaucher's disease, SEC, Growth, Cancer, OTCQB, Security (finance)

Subject to certain limitations, each outstanding share of Series D Preferred Stock will have 1,000,000 votes per share (or 1,000 votes per one one-thousandth of a share of Series D Preferred Stock).

Key Points: 
  • Subject to certain limitations, each outstanding share of Series D Preferred Stock will have 1,000,000 votes per share (or 1,000 votes per one one-thousandth of a share of Series D Preferred Stock).
  • The Series D Preferred Stock will be uncertificated, and no shares of Series D Preferred Stock will be transferable by any holder thereof except in connection with a transfer by such holder of any shares of the Companys common stock held by such holder.
  • In that case, a number of one one-thousandths of a share of Series D Preferred Stock equal to the number of shares of the Companys common stock to be transferred by such holder would be transferred to the transferee of such shares of common stock.
  • Further details regarding the Series D Preferred Stock will be contained in a report on Form 8-K to be filed by the Company with the Securities and Exchange Commission.

Gain Therapeutics, Inc. Reports First Quarter 2022 Financial Results and Business Update

Retrieved on: 
Monday, May 16, 2022
Clinical trial, Autophagosome, Research, Survival, Nasdaq, GD, Gaucher's disease, Neurodegeneration, Patient, GBA1, Alpha-synuclein, Information technology, Trial of the century, Therapy, Macrophage, XXVII, AD, Hope, Neuron, Brain, CNS, Lysosome, Conference, GBA, Lead, Parkinson, Program, WORLD, Liver support system, Allosteric regulation, PD, GAAP, Drug discovery, Potency (pharmacology), GLOBE, Medical device, Pharmaceutical industry, Aquaculture, Airline, WT, Gaucher

BETHESDA, Md., May 16, 2022 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc. (Nasdaq: GANX) (“Gain”, or the “Company”), a biotechnology company transforming drug discovery with its proprietary computational discovery platform identifying novel allosteric binding sites and creating small molecule treatments, today announced its financial results for the first quarter ended March 31, 2022, and highlighted recent corporate accomplishments.

Key Points: 
  • This has been a productive quarter for Gain, as we continued to demonstrate the broad reaching potential of our SEE-Tx platform, said Eric Richman, Chief Executive Officer of Gain Therapeutics.
  • Our strategy of exploring previously unidentified allosteric binding sites and targeting them for therapeutic benefit has proved promising.
  • In March, Gain presented, Targeting Glucocerebrosidase with Structurally Targeted Allosteric Regulators Corrects Abnormal Phenotypes in Models of Parkinsons Disease.
  • Cash and cash equivalents were $34.34 million as of March 31, 2022 compared to $36.88 million at December 31, 2021.

Gain Therapeutics Presents Positive Preclinical Data On Its Gaucher Disease Program at IWGGD Symposium

Retrieved on: 
Wednesday, May 11, 2022
Population, Brain, Nasdaq, GBA1, GBA, GLOBE, CNS, The Michael J. Fox Foundation, Lysosomal storage disease, Lysosome, MJFF, Time, Drug discovery, Gaucher's disease, Patient, Neuron, Lists of diseases, Macrophage, Glycogen storage disease, GD, Disease, Enzyme replacement therapy, Traffic barrier, Protein, Therapy, Pharmaceutical industry, Medical imaging, Gaucher, NGD

The results were highlighted in an oral presentation at the 1st IWGDD Symposium being held May 8-11, 2022, in the Netherlands.

Key Points: 
  • The results were highlighted in an oral presentation at the 1st IWGDD Symposium being held May 8-11, 2022, in the Netherlands.
  • Gaucher disease is traditionally classified according to one of three types, of which Gaucher disease type 1 is a non-neuronopathic form, whereas Gaucher disease types 2 and 3 are considered neuronopathic Gaucher disease (nGD) with early onset brain degeneration that gets progressively worse over time.
  • Gain Therapeutics, Inc. is transforming the drug discovery paradigm with structurally targeted allosteric regulators identified with its proprietary computational discovery platform SEE-Tx.
  • Gains lead program in Parkinsons disease has been awarded funding support from The Michael J.

Freeline Reports First Quarter 2022 Financial Results and Business Highlights

Retrieved on: 
Tuesday, May 10, 2022
Haemophilia B, Form 6-K, COVID-19, Data monitoring committee, Research and development, Acceleration, Three Months Gone, Forward-looking statement, Flood, U.S. Securities and Exchange Commission, Nasdaq, Failure, GLOBE, Fabry disease, Company, AAV, Physician, Safety, Earthquake, Gaucher's disease, Patient, Doctor of Philosophy, Bleeding, Terrorism, Clinical trial, Program, D&O, Administration, Haemophilia A, Lists of disasters, Private Securities Litigation Reform Act, CMO, Science, Fire, Transgene, EDGAR, SEC, Terminology, Infection, Factor IX, Research, Management, Pharmaceutical industry, Vaccine, Renewable energy, Gaucher, Haemophilia, Daimler Freeline

LONDON, May 10, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”) today reported financial results for the first quarter of 2022 and provided a business update.

Key Points: 
  • Phase 1/2 clinical programs in Hemophilia B, Gaucher disease and Fabry disease on track to deliver 2022 milestones
    LONDON, May 10, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the Company or Freeline) today reported financial results for the first quarter of 2022 and provided a business update.
  • The first cohort was completed in April 2022 following the dosing of the first patient in March 2022.
  • Following the appointment of Henning Stennicke, PhD, as Chief Scientific Officer in March 2022, Paul Schneider will be joining Freeline as its new Chief Financial Officer on May 16, 2022, as announced in April 2022.
  • For further information, please reference the Companys reports and documents filed with the U.S. Securities and Exchange Commission (the SEC).

Voyager Therapeutics Reports First Quarter 2022 Financial and Operating Results

Retrieved on: 
Wednesday, May 4, 2022
D, Parkinson's disease, NHP, Neurocrine Biosciences, Gaucher's disease, Genzyme, Annual report, RNA interference, Brain, CEO, Risk, Library, GBA1, Company, NYSE, Private Securities Litigation Reform Act, AAV, Patient, FDA, Neoplasm, Robin, Lists of diseases, Tauopathy, Tropism, Research, Therapy, U.S. Securities and Exchange Commission, Capsid, Senior, Mouse, RNA, Security (finance), Survival, Cardiac muscle, B cell, Islamic finance products, services and contracts, Exercise, Board, COVID-19, Microglia, Nasdaq, GLOBE, CNS, SOD1, NVS, ALS, Conference, Traffic barrier, G&A, Pathology, HER2, Vaccine, Medical device, Pharmaceutical industry, Management, Online gambling, Voyager, Novartis, Pfizer

Collaboration Revenues: Voyager had collaboration revenue of $0.7 million for the first quarter of 2022, compared to $6.5 million for the same period in 2021.

Key Points: 
  • Collaboration Revenues: Voyager had collaboration revenue of $0.7 million for the first quarter of 2022, compared to $6.5 million for the same period in 2021.
  • Voyager Therapeutics (Nasdaq: VYGR) is leading the next generation of AAV gene therapy to unlock the potential of the modality to treat devastating diseases.
  • voyagertherapeutics.com LinkedIn Twitter
    Voyager Therapeutics is a registered trademark, and TRACER is a trademark, ofVoyager Therapeutics, Inc.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected.