Gaucher's disease

Freeline Announces $26.1 Million Registered Direct Offering of American Depositary Shares

Retrieved on: 
Friday, March 11, 2022
Nasdaq, Safety, United Kingdom, Prospectus, Order, Private Securities Litigation Reform Act, Company, Research, U.S. Securities and Exchange Commission, Financial services, Failure, EEA, Transgene, ADS, Investment, Person, European Union (Withdrawal) Act 2018, ADSS, Infection, GLOBE, Gaucher, Sale, Patient, European Economic Area, Security (finance), Form, Suite, Finance, EDGAR, Urban economics, American depositary receipt, Terminology, COVID-19, Fabry disease, AAV, Gaucher's disease, Financial Services and Markets Act 2000, SEC, SEC filing, Haemophilia B, Forward-looking statement, Pharmaceutical industry, EU

The Company has engaged Wedbush Securities Inc. as its financial advisor in connection with the offering.

Key Points: 
  • The Company has engaged Wedbush Securities Inc. as its financial advisor in connection with the offering.
  • The securities described above will be offered by the Company pursuant to an effective shelf registration statement on Form F-3 (File No.
  • 333-259444) previously filed with the Securities and Exchange Commission (the SEC) on September 10, 2021 and declared effective by the SEC on September 22, 2021.
  • The securities may be offered only by means of a written prospectus and prospectus supplement that form a part of the registration statement.

CytRx Announces Integration of Wholly Owned Subsidiary Centurion BioPharma and Corporate Governance Updates

Retrieved on: 
Wednesday, March 9, 2022
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CytRx Corporation (OTCQB: CYTR) (CytRx or the Company), a biopharmaceutical innovator focused on research and development of life-saving cancer therapeutics, today announced that the Company is absorbing its wholly-owned subsidiary Centurion BioPharma Corporation (Centurion).

Key Points: 
  • CytRx Corporation (OTCQB: CYTR) (CytRx or the Company), a biopharmaceutical innovator focused on research and development of life-saving cancer therapeutics, today announced that the Company is absorbing its wholly-owned subsidiary Centurion BioPharma Corporation (Centurion).
  • CytRx thanks Dr. Ignarro and Mr. Kriegsman for their many years of service and wishes them well in their future pursuits.
  • A new independent director will be added to the Board at or before the 2022 Annual Meeting of Shareholders.
  • All forward-looking statements are based upon information available to the CytRx on the date the statements are first published.

Freeline Announces First Patient Dosed in the Phase 1/2 B-LIEVE Dose-Confirmation Trial in Hemophilia B

Retrieved on: 
Wednesday, March 9, 2022
Nasdaq, Safety, Sex linkage, Private Securities Litigation Reform Act, Patient, Company, Form 6-K, Research, Haemophilia, Failure, Factor IX, Transgene, Securities and Exchange Commission (Philippines), VIII, MD, Infection, Woman, GLOBE, FIX, Blood, EDGAR, Terminology, Man, COVID-19, Fabry disease, AAV, Gaucher's disease, ECLIPSE, Haemophilia B, Forward-looking statement, Vaccine, Pharmaceutical industry, EU, Daimler Freeline

LONDON, March 09, 2022 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) (the “Company” or “Freeline”) today announced that the first patient was dosed in its Phase 1/2 B-LIEVE dose-confirmation clinical trial of FLT180a for the treatment of hemophilia B, a debilitating genetic bleeding disorder caused by a deficiency in the clotting factor IX protein. FLT180a uses a potent and proprietary adeno-associated virus vector capsid (AAVS3) to deliver the functioning gene of a variant of human factor IX and restore normal blood clot formation.

Key Points: 
  • In addition, this is the first patient Freeline has dosed in the U.S., which affirms our commitment to diversify our clinical trial footprint and build resiliency into our programs.
  • The Company expects to report initial safety and biomarker data from the first dose cohort of the B-LIEVE trial in the first half of 2022.
  • The Company has clinical programs in hemophilia B, Fabry disease, and Gaucher disease Type 1.
  • FLT180a was 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.

Voyager Therapeutics Reports Fourth Quarter and Full Year 2021 Financial and Operating Results

Retrieved on: 
Tuesday, March 8, 2022
Industry, Islamic finance products, services and contracts, Neurocrine Biosciences, Security (finance), RNA, Lists of diseases, D, Board, COVID-19, Cyprus Securities and Exchange Commission, Executive Committee, Nasdaq, ALS, NVS, Private Securities Litigation Reform Act, NHP, Robin, Gaucher's disease, Tropism, IV, LinkedIn, FDA, Therapy, Brain, Company, AAV, R, GBA1, Tissue, Neoplasm, Conference call, Risk, Research, COVID, Director, Investor, Annual report, Primate, NYSE, CNS, Technology, Exercise, PFE, Prevalence, Toxic shock syndrome, Income, Genzyme, Board of directors, AbbVie, SOD1, Library, Senior, Conference, Cardiac muscle, Traffic barrier, GLOBE, Management, Pharmaceutical industry, Vaccine, Pfizer, Voyager, G&A, Novartis

ET

Key Points: 
  • ET
    CAMBRIDGE, Mass., March 08, 2022 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a gene therapy company developing life-changing treatments and next-generation adeno-associated virus (AAV) capsids, today reported fourth quarter and full year 2021 financial and operating results.
  • Collaboration Revenues: Voyager had collaboration revenue of $28.1 million for the fourth quarter of 2021, compared to $6.5 million for the same period in 2020.
  • ET to provide and discuss the fourth quarter and full year 2021 financial and operating results and the Novartis license option agreement.
  • All forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected.

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

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

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.

Global Gaucher's Disease Market and Competitive Landscape - 2021 - ResearchAndMarkets.com

Retrieved on: 
Friday, March 4, 2022
Genetics, Health, Clinical Trials, Disease, Gaucher's disease, Gaucher, Research report, Epidemiology, Research, Competitive landscape, G7, Prevalence, Dietary supplement, Pharmaceutical industry

The "Global Gaucher's Disease Market and Competitive Landscape - 2021" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Gaucher's Disease Market and Competitive Landscape - 2021" report has been added to ResearchAndMarkets.com's offering.
  • The research report provides comprehensive insights into Gaucher's Disease pipeline, epidemiology, market valuations, drug sales, market forecast, drug forecasts, and market shares.
  • This research covers the following - Gaucher's Disease treatment options, Gaucher's Disease late-stage clinical trials pipeline, Gaucher's Disease prevalence by countries, Gaucher's Disease market size and forecast by countries, key market events and trends, drug sales and forecast by countries, and market shares by countries.
  • Gaucher's Disease pipeline: Find out drugs in clinical trials for the treatment of Gaucher's Disease by development phase 3, phase 2, by pharmacological class and company
    Gaucher's Disease drugs: Identify key drugs marketed and prescribed for Gaucher's Disease in the US, including trade name, molecule name, and company
    Gaucher's Disease market valuations: Find out the market size for Gaucher's Disease drugs in 2020 by countries.

Global Homocystinuria Market Analysis/Forecast Report 2021-2028: Opportunities with Pyridoxine as a First-Line Agent for Homocystinuria - ResearchAndMarkets.com

Retrieved on: 
Monday, February 28, 2022
Health, Other Health, Lists of diseases, Diagnosis, Phenylketonuria, Degenerative disease, Growth, Prevalence, Laboratory, Betaine, Awareness, National library, Gaucher's disease, COVID-19, Pyridoxine, Method, Genetic testing, Hospital, Homocystinuria, Disorder, Treatment, Glycogen, National Library of Medicine classification, Academy, Geography, CAGR, Research, Pharmaceutical industry, Medical device

The growth of the market is attributed to the rising deficiencies of vitamin B6 and vitamin B12 and the growing prevalence of inherited metabolic disorders.

Key Points: 
  • The growth of the market is attributed to the rising deficiencies of vitamin B6 and vitamin B12 and the growing prevalence of inherited metabolic disorders.
  • However, the lack of awareness about various rare disorders and inherited metabolic disorders hampers the market growth.
  • Lysosomal, storage disorder, Gaucher disease, phenylketonuria, glycogen storage diseases, mitochondrial disorders, and homocystinuria are a few inherited metabolic disorders.
  • Thus, the increase in the prevalence of inherited metabolic disorders is expected to boost the growth of the homocystinuria market worldwide.

CytRx Comments on Orphazyme's Update on European Union Regulatory Review of Arimoclomol in Niemann-Pick Disease Type C

Retrieved on: 
Thursday, February 24, 2022
Biotechnology, General Health, Pharmaceutical, Health, CytRx, Therapy, Niemann–Pick disease, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Head, European Medicines Agency, NASDAQ, ImmunityBio, NPC, Marketing, Gaucher's disease, Company, Glioblastoma, Pancreatic cancer, Review, Orientation, Food, News, Neurodegeneration, Committee for Medicinal Products for Human Use, OTCQB, Cancer, EMA, MAA, Patient, Union, Technology, Committee, CHMP, Research, Marketing Authorisation Application, Pharmaceutical industry

CytRx licensed arimoclomol to Orphazyme in exchange for milestone payments and royalties.

Key Points: 
  • CytRx licensed arimoclomol to Orphazyme in exchange for milestone payments and royalties.
  • The trend vote indicates that the CHMP's current orientation is to not approve arimoclomol when it convenes by the end of March 2022.
  • In addition, CytRxs drug candidate, arimoclomol, was sold to Orphazyme A/S in exchange for milestone payments and royalties.
  • Orphazyme is developing arimoclomol in two indications, including Niemann-Pick disease Type C (NPC), and Gaucher disease.

BlueAllele Corporation Secures Second Patent from USPTO Strengthening the Company's Position in Targeted Insertion and Gene Correction Technologies

Retrieved on: 
Tuesday, February 22, 2022
Central nervous system, Therapy, Hurler syndrome, Retinitis pigmentosa, Patent, Method, Global, Retina, AAV, Gaucher's disease, Haemophilia, Industry, Genome, Company, USPTO, Lists of diseases, Acceleration, Hunter syndrome, NHEJ, ALS, United States Patent and Trademark Office, Fabry disease, Liver, 1836 U.S. Patent Office fire, Technology, Glycogen, Research, Gene editing, Vaccine, Antibiotics, Pharmaceutical industry

11,254,930 (the '930 Patent) related to the palindromic, bi-directional structure of repair templates for targeted insertion and gene correction using the non-homologous end joining (NHEJ) pathway.

Key Points: 
  • 11,254,930 (the '930 Patent) related to the palindromic, bi-directional structure of repair templates for targeted insertion and gene correction using the non-homologous end joining (NHEJ) pathway.
  • BlueAllele's previously granted US patent 11,091,756 (the '756 Patent) includes claims covering methods using palindromic, bi-directional repair templates for gene correction.
  • Both the '930 Patent and the '756 Patent are exclusively owned by BlueAllele Corporation and provide broad coverage for the development and advancement of human gene correction technologies.
  • BlueAllele Corporation is a biotechnology company committed to advancing transformational gene correction technologies including its patented PALIDON repair templates and on the research and development of precision gene correction technologies.

CytRx Highlights Orphazyme's Update on Planned NDA Resubmission for Arimoclomol in Niemann-Pick Disease Type C

Retrieved on: 
Monday, February 14, 2022
Oncology, Health, FDA, Research, Science, Pharmaceutical, Biotechnology, NASDAQ, Patient, Food, Cancer, ImmunityBio, European Medicines Agency, Company, Neurodegeneration, OTCQB, Endpoint security, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Therapy, Niemann–Pick disease, Gaucher's disease, NPC, EU, CytRx, Committee, European Directive on Traditional Herbal Medicinal Products, FDA, NDA, Research, Type, Committee for Medicinal Products for Human Use, Pharmaceutical industry

CytRx licensed arimoclomol to Orphazyme in exchange for milestone payments and royalties.

Key Points: 
  • CytRx licensed arimoclomol to Orphazyme in exchange for milestone payments and royalties.
  • Orphazyme plans to request the Type C Meeting in Q2 2022.
  • CytRx will continue to share publicly available updates as they relate to the Company's licensing deal with Orphazyme.
  • Orphazyme is developing arimoclomol in two indications, including Niemann-Pick disease Type C (NPC), and Gaucher disease.