Gaucher

AVROBIO Announces Agreement to Sell Cystinosis Gene Therapy Program for $87.5 Million

Retrieved on: 
Monday, May 22, 2023
Health, Stem Cells, Genetics, Other Health, Clinical Trials, Pharmaceutical, Biotechnology, TD, Glycogen storage disease type II, Sale, Cystinosis, Gaucher, AVRO, Wells Fargo, CFO, HSC, Hunter syndrome, DSM-IV codes, Pharmaceutical industry, Financial adviser, Novartis

(Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today announced an agreement to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of 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 an agreement to sell its investigational hematopoietic stem cell (HSC) gene therapy program for the treatment of cystinosis to Novartis for $87.5 million in cash.
  • Pursuant to the terms of an asset purchase agreement, Novartis will pay AVROBIO $87.5 million in cash at closing, in consideration for the sale and transfer of certain assets related to the cystinosis program.
  • In addition, AVROBIO has exclusively licensed to Novartis certain other assets, know-how and other intellectual property related to AVROBIO’s gene therapy platform for use in cystinosis.
  • To support the transition of the program, AVROBIO also has agreed to provide under a separate agreement certain transition, knowledge transfer and other related services.

AVROBIO Reports First Quarter 2023 Financial Results and Provides Business Update

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Thursday, May 11, 2023
Health, Stem Cells, Neurology, Genetics, Clinical Trials, Pharmaceutical, Biotechnology, GMP, VCN, Gaucher's disease, Central nervous system, University, Gene, Clinical trial, Glycogen, MPS II, Skin, Glycogen storage disease type II, MPS, CNS, Hunter syndrome, Gaucher, AVRO, HSPC, Patient, Physician, Annual general meeting, Cell, CFO, American Society of Gene and Cell Therapy, HSC, Innovation, UOM, FDA, Enzyme replacement therapy, Syndrome, Research, Safety, Society, GI, Cystine, DSM-IV codes, MHRA, Pharmaceutical industry, Medical device, Cryptocurrency

(Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2023, and provided a business update.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the first quarter ended March 31, 2023, and provided a business update.
  • Plan to initiate Guard3, a global registrational Phase 2/3 trial for GD3, in the second half of 2023, subject to regulatory alignment.
  • AVROBIO reported a net loss of $25.0 million for the first quarter of 2023 as compared to a net loss of $29.8 million for the comparable period in 2022.
  • Based on AVROBIO’s current operating plan, AVROBIO expects its cash and cash equivalents as of March 31, 2023, will enable AVROBIO to fund its operating expenses and capital expenditure requirements into the first quarter of 2024.

Former Pfizer Legal Counsel Discusses Gene Therapy with CancerVAX CEO Ryan Davies

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Thursday, April 20, 2023
Diamond, Business development, University, Therapy, Senior, General counsel, University of Nairobi, Gene editing, Assistant General Counsel of the Navy (Research, Development and Acquisitions), Cancer, New York State Bar Association, Milbank, LEHI, Associate, Assistant, Gaucher, Barclays, Patient, Corporation, William, Mergers and acquisitions, Conditional sentence, William and Mary, Pharmaceutical industry, New York University, Axiom, Pfizer

LEHI, Utah, April 20, 2023 (GLOBE NEWSWIRE) -- CancerVAX, developer of a breakthrough universal cancer vaccine that will use the body’s immune system to fight cancer, reported that the Company’s CEO, Ryan Davies, recently discussed gene therapy’s role in the treatment of chronic diseases with Stephen Diamond, the former legal counsel for Pfizer.

Key Points: 
  • Stephen Diamond, Senior Vice President, General Counsel, and Secretary of Freeline, discussed his unique path from the legal and business worlds to gene therapy and next-generation therapeutics.
  • LEHI, Utah, April 20, 2023 (GLOBE NEWSWIRE) -- CancerVAX, developer of a breakthrough universal cancer vaccine that will use the body’s immune system to fight cancer, reported that the Company’s CEO, Ryan Davies, recently discussed gene therapy’s role in the treatment of chronic diseases with Stephen Diamond, the former legal counsel for Pfizer.
  • He formerly served as Assistant General Counsel and Senior Corporate Counsel of Mergers & Acquisitions for Pfizer, while concurrently serving as a Corporate Attorney of Axiom.
  • “I was very excited to speak with Stephen,” said CancerVAX CEO Ryan Davies.

Freeline Reports Full Year 2022 Financial Results and Provides Corporate Update

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Tuesday, April 4, 2023
Workforce, University College London, Gaucher's disease, Half-life, Hematology, Clinical trial, Doctor of Philosophy, Bone pain, Sale, Parini, MD, Patient, Administration, Webcast, CFO, Research and development, R, Research, Fabry disease, Glucocerebrosidase, Therapy, Safety, Disease, Cryptocurrency, Pharmaceutical industry, Management, Fabry, Gaucher, Daimler Freeline

ET

Key Points: 
  • ET
    LONDON, April 04, 2023 (GLOBE NEWSWIRE) -- Freeline Therapeutics Holdings plc (Nasdaq: FRLN) today reported financial results for the full year ended December 31, 2022 and provided a corporate update.
  • Freeline also announced the appointment of its Chief Financial Officer Paul M. Schneider to the company’s Board of Directors.
  • Mr. Schneider has an impressive track record of financial stewardship, including corporate strategy and execution, financial planning, analysis and reporting, and investor relations.
  • His addition to the board will be instrumental as we work to advance FLT201 in Gaucher disease and strengthen our financial position.

Oral Clinical Nutrition Global Market Report 2023: Sector to Reach Grow by Over 5% Annually Through 2033 - ResearchAndMarkets.com

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Monday, March 27, 2023
Fitness & Nutrition, Health, Male, Chronic kidney disease, Obesity, Surgeon, Eosinophilic esophagitis, Food, National Cancer Institute (Egypt), Prevalence, Nutritional deficiency eczema, Diabetes, Malnutrition, Pediatrics, Organization, Cancer, Woman, FPIES, CKD, USD, Sale, Government, Micronutrient, Projection, Hypertension, Somatic symptom disorder, Vitamin, Incidence, Alzheimer's disease, Protein, Apathy, Fatigue, Hospital, Pain management, Patient, European, Economic Cooperation Organization, Growth, Nutricia, Region, Development, National Cancer Institute, GI, Environment, NIH, Female, Amino acid-based formula, Malabsorption, Dietary supplement, Pharmaceutical industry, Dysphagia, Gaucher

The "Oral Clinical Nutrition Market by Stage, Indication Sales Channel Outlook and by Region - Global Forecast to 2023-2033" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Oral Clinical Nutrition Market by Stage, Indication Sales Channel Outlook and by Region - Global Forecast to 2023-2033" report has been added to ResearchAndMarkets.com's offering.
  • The oral clinical nutrition market size is estimated to be USD 15,358.7 million in 2022 and is expected to witness a CAGR of 5.42% during the forecast period 2023-2033.
  • However, uncertain perceptions of therapeutic nutrition and a decline in birth rates are anticipated to hinder the market growth.
  • clinical nutrition plays a significant role in avoiding harmful effects for patients during procedures and allowing surgeons to operate in a controlled environment.

AVROBIO Reports Fourth Quarter and Fiscal Year 2022 Financial Results and Provides Business Update

Retrieved on: 
Thursday, March 23, 2023
Health, Other Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, GMP, Tissue, VCN, University of California, San Diego Performance-Based Skills Assessment, Gaucher's disease, Enteropathy, University, Safety, Health care, Food, CMC, Aes, Clinical trial, MPS II, Skin, Glycogen storage disease, Cerebrospinal fluid, MPS, CNS, IDS, Cystinosis, AVRO, ERT, HSPC, Patient, Physician, Risk, Medicines and Healthcare products Regulatory Agency, Liver, CSF, HSC, UOM, FDA, Syndrome, Enzyme replacement therapy, Lymphadenopathy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Hunter syndrome, GI, Cystine, DSM-IV codes, MHRA, Disease, Pharmaceutical industry, Vaccine, Gaucher

(Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2022 and provided a business update.

Key Points: 
  • (Nasdaq: AVRO), a leading clinical-stage gene therapy company working to free people from a lifetime of genetic disease, today reported financial results for the fourth quarter and year ended Dec. 31, 2022 and provided a business update.
  • “2022 was a transformative year for AVROBIO, topped in December with a robust clinical and regulatory update for our Gaucher disease program.
  • Safety data to date from GD1 and GD3 patients indicate no adverse events (AEs) related to drug product.
  • No major chemistry, manufacturing and controls (CMC) changes are anticipated for AVROBIO’s plato® gene therapy platform as the company prepares to enter a registrational trial for GD3.

Ten new companies enter BioInnovation Institute's Venture Lab program

Retrieved on: 
Wednesday, March 1, 2023
Parkinson's disease, Therapy, Metastasis, BII, Detergent, Health, Gaucher, Biomass, Drug development, Research, Patient, Cosmetics, Conditional sentence, Neoplasm, Machine learning, Gaucher's disease, Data science, EUR, RNA, Pharmaceutical industry, Drug

COPENHAGEN, Denmark, March 1, 2023 /PRNewswire/ -- BioInnovation Institute (BII), an international non-profit foundation incubating and accelerating world-class life science research, today announces that ten new companies have entered its Venture Lab acceleration program for early-stage companies.

Key Points: 
  • COPENHAGEN, Denmark, March 1, 2023 /PRNewswire/ -- BioInnovation Institute (BII), an international non-profit foundation incubating and accelerating world-class life science research, today announces that ten new companies have entered its Venture Lab acceleration program for early-stage companies.
  • In becoming a part of the Venture Lab program, the early-stage companies also get an exclusive opportunity to apply for EUR 1.4M in follow-up funding through BII's Venture House program.
  • Bobby Soni, Chief Business Officer at BioInnovation Institute, said: "We are pleased to welcome this new cohort of early-stage companies working across the bioindustrials, therapeutics, women's health and health tech industries to the Venture Lab program.
  • The new companies BII has accepted into the Venture Lab acceleration program are:
    Agoprene develops furniture foam from biomass to help the furniture industry reduce the need for petrochemicals.

Ten new companies enter BioInnovation Institute's Venture Lab program

Retrieved on: 
Wednesday, March 1, 2023
Parkinson's disease, Therapy, Metastasis, BII, Detergent, Health, Gaucher, Biomass, Drug development, Research, Patient, Cosmetics, Conditional sentence, Neoplasm, Machine learning, Gaucher's disease, Data science, EUR, RNA, Pharmaceutical industry, Drug

COPENHAGEN, Denmark, March 1, 2023 /PRNewswire/ -- BioInnovation Institute (BII), an international non-profit foundation incubating and accelerating world-class life science research, today announces that ten new companies have entered its Venture Lab acceleration program for early-stage companies.

Key Points: 
  • COPENHAGEN, Denmark, March 1, 2023 /PRNewswire/ -- BioInnovation Institute (BII), an international non-profit foundation incubating and accelerating world-class life science research, today announces that ten new companies have entered its Venture Lab acceleration program for early-stage companies.
  • In becoming a part of the Venture Lab program, the early-stage companies also get an exclusive opportunity to apply for EUR 1.4M in follow-up funding through BII's Venture House program.
  • Bobby Soni, Chief Business Officer at BioInnovation Institute, said: "We are pleased to welcome this new cohort of early-stage companies working across the bioindustrials, therapeutics, women's health and health tech industries to the Venture Lab program.
  • The new companies BII has accepted into the Venture Lab acceleration program are:
    Agoprene develops furniture foam from biomass to help the furniture industry reduce the need for petrochemicals.

Gain Therapeutics Presents New Preclinical Data from its Gaucher Disease Program at the 19th Annual WORLDSymposium

Retrieved on: 
Monday, February 27, 2023
Gaucher, Trial of the century, Inflammation, CBE, Therapy, Mouse, Drug discovery, GBA1, Patient, Pharmaceutical industry, Glucocerebrosidase

BETHESDA, Md., Feb. 27, 2023 (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, presented new pre-clinical data from its Gaucher disease program in a poster presentation at the 19th Annual WORLDSymposium held February 22–26, 2023, in Orlando, Florida.

Key Points: 
  • BETHESDA, Md., Feb. 27, 2023 (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, presented new pre-clinical data from its Gaucher disease program in a poster presentation at the 19th Annual WORLDSymposium held February 22–26, 2023, in Orlando, Florida.
  • The data generated in an animal model of neuronopathic Gaucher disease (nGD) show that GT-02329 restores β-glucocerebrosidase (GCase) activity, depletes accumulation of toxic lipid substrates, reduces neuroinflammation and improves neuromuscular function.
  • “These data are a further validation of the disease-modifying potential of our GCase-targeting small molecule compounds in diseases caused by mutations of the GBA1 gene, including for the treatment of nGD and GBA1 Parkinson’s disease,” said Matthias Alder, Chief Executive Officer of Gain.
  • CBE is a covalent inhibitor that causes a reduction in GCase activity by reacting with the catalytic site of GCase and inactivating the GCase enzyme, which results in neurological features that occur in nGD patients, including the accumulation of the toxic lipid substrate glucosylsphingosine.

AceLink Presents Preclinical Data on AL00804 at WORLD Symposium

Retrieved on: 
Thursday, February 23, 2023
Biotechnology, Infectious Diseases, Health, Genetics, Pharmaceutical, GL1, DSM-IV codes, Therapy, Doctor of Philosophy, Cincinnati Children's Hospital Medical Center, GSL, Brain, Patient, CNS, Gaucher's disease, Disney Star, Traffic barrier, Pharmaceutical industry, Medical imaging, Vaccine, Gaucher

AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today presented data on the development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid (GSL) storage diseases.

Key Points: 
  • AceLink Therapeutics, Inc. (AceLink), an innovative biopharmaceutical company developing transformative therapies for genetic diseases, today presented data on the development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid (GSL) storage diseases.
  • “We are pleased with the efficacy of AL00804 in reducing symptoms of CNS disease in several preclinical models,” said Jerry Shen, PhD, Chief Executive Officer and Founder of AceLink.
  • AL00804 is highly brain penetrant that efficiently reduced brain GL1 and lyso-GL1 accumulation in preclinical disease models.
  • Abstract Title: Development of AL00804, a novel brain penetrant glucosylceramide synthase inhibitor, to treat Gaucher disease and other neuronopathic glycosphingolipid storage diseases