Rare diseases

AVROBIO to Present New Clinical and Preclinical Data at Upcoming American Society of Gene & Cell Therapy (ASGCT) Annual Meeting, May 12-15, 2020

Retrieved on: 
Tuesday, May 5, 2020
Health, FDA, Genetics, Clinical trials, Research, Science, Biotechnology, Inborn errors of metabolism, Branches of biology, Health, Rare diseases, Lysosomal storage diseases, Drug discovery, Applied genetics, Biotechnology, Gene therapy, Fabry disease, Therapy, Cystinosis

The oral presentations will include data updates for its investigational programs for Fabry disease and cystinosis, as well as preclinical data for its investigational program for Pompe disease.

Key Points: 
  • The oral presentations will include data updates for its investigational programs for Fabry disease and cystinosis, as well as preclinical data for its investigational program for Pompe disease.
  • Our mission is to free people from a lifetime of genetic disease with a single dose of gene therapy.
  • AVROBIO is powered by the plato gene therapy platform, our foundation designed to scale gene therapy worldwide.
  • AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

Amicus Therapeutics Announces Upcoming Presentations at the American Society of Gene & Cell Therapy 23rd Annual Meeting

Retrieved on: 
Tuesday, May 5, 2020
Branches of biology, Medicine, Inborn errors of carbohydrate metabolism, Biology, Rare diseases, Hepatology, Lysosomal storage diseases, Glycogen storage disease type II, Acid alpha-glucosidase, Pompe, Gene therapy, Weakness

Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation.

Key Points: 
  • Preclinical data from its Pompe gene therapy program, which Amicus is developing with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania, will be presented as an oral presentation.
  • Reduced or absent levels of GAA leads to accumulation of glycogen in cells, which results in the clinical manifestations of Pompe disease.
  • The disease can be debilitating and is characterized by severe muscle weakness that worsens over time.
  • You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof.

Bronchial Dysplasia Pipeline Insight, 2020: Therapeutic Assessment, Unmet Needs, Products, Drugs, Companies - ResearchAndMarkets.com

Retrieved on: 
Friday, May 1, 2020
Health, Other Health, Managed care, Clinical trials, General Health, Pharmaceutical, Rare diseases

Bronchial Dysplasia Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Bronchial Dysplasia market.

Key Points: 
  • Bronchial Dysplasia Pipeline Insight, 2020 outlays comprehensive insights of present clinical development scenario and growth prospects across the Bronchial Dysplasia market.
  • A detailed picture of the Bronchial Dysplasia pipeline landscape is provided, which includes the disease overview and Bronchial Dysplasia treatment guidelines.
  • The assessment part of the report embraces in-depth Bronchial Dysplasia commercial assessment and clinical assessment of the Bronchial Dysplasia pipeline products from the pre-clinical developmental phase to the marketed phase.
  • Different therapeutic candidates segmented into early-stage, mid-stage and late stage of development for the Bronchial Dysplasia treatment.

Recessive Dystrophic Epidermolysis (RDEB) Pipeline Insight, 2020 - Therapeutic Assessment, Unmet Needs, Products, Drugs, Companies - ResearchAndMarkets.com

Retrieved on: 
Friday, May 1, 2020
Health, Other Health, Other Science, Managed care, Research, Science, Pharmaceutical, Genodermatoses, Rally of the Ecologists of Burkina, Rare diseases, Health, Scioderm

This report outlays comprehensive insights of present clinical development scenario and growth prospects across the Recessive Dystrophic Epidermolysis (RDEB) market.

Key Points: 
  • This report outlays comprehensive insights of present clinical development scenario and growth prospects across the Recessive Dystrophic Epidermolysis (RDEB) market.
  • A detailed picture of the Recessive Dystrophic Epidermolysis (RDEB) pipeline landscape is provided, which includes the disease overview and Recessive Dystrophic Epidermolysis (RDEB) treatment guidelines.
  • The assessment part of the report embraces in-depth Recessive Dystrophic Epidermolysis (RDEB) commercial assessment and clinical assessment of the Recessive Dystrophic Epidermolysis (RDEB) pipeline products from the pre-clinical developmental phase to the marketed phase.
  • Different therapeutic candidates segmented into early-stage, mid-stage and late stage of development for the Recessive Dystrophic Epidermolysis (RDEB) treatment.

Seelos Therapeutics Receives Orphan Drug Designation for SLS-005 (Trehalose) in Sanfilippo Syndrome

Retrieved on: 
Thursday, April 30, 2020
Health, Articles, Pharmaceuticals policy, Food and Drug Administration, Life sciences, Rare diseases, 97th United States Congress, Drug policy of the United States, Orphan Drug Act, Orphan drug, Spinocerebellar ataxia, Fast track

NEW YORK, April 30, 2020 (GLOBE NEWSWIRE) -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced it has been granted Orphan Drug Designation (ODD) for SLS-005 in Sanfilippo syndrome from U.S. Food and Drug Administration (FDA).

Key Points: 
  • NEW YORK, April 30, 2020 (GLOBE NEWSWIRE) -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced it has been granted Orphan Drug Designation (ODD) for SLS-005 in Sanfilippo syndrome from U.S. Food and Drug Administration (FDA).
  • SLS-005 was previously granted Orphan Drug Designation from the FDA and European Medicines Agency (EMA) for Spinocerebellar Ataxia Type 3 (SCA3) and Oculopharyngeal Muscular Dystrophy (OPMD) as well as Fast Track designation for OPMD.
  • The Orphan Drug Act (ODA) provides for granting special status to a drug or biological product (drug) to treat a rare disease or condition upon request of a sponsor.
  • Orphan designation qualifies the sponsor of the drug for various development incentives of the ODA, including tax credits for qualified clinical testing.

US Societal Economic Burden of an Inherited Kidney Disease Estimated at $7-10 Billion

Retrieved on: 
Thursday, April 30, 2020
Kidney diseases, Rare diseases, Autosomal dominant polycystic kidney disease, Channelopathies, Health, Medical specialties, Kidney, Pharmaceutical industry

It is characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure.

Key Points: 
  • It is characterized by progressive development of renal cysts and numerous extra-renal manifestations, eventually leading to kidney failure.
  • "The economic burden associated with rare diseases is often overlooked by physicians, payers, and regulators," said Myrlene Sanon Aigbogun, Director, Health Economics and Outcomes Research, Otsuka Pharmaceutical Development & Commercialization.
  • "By publishing this study, we hope to increase awareness of the economic burden associated with ADPKD, and show that the costs associated with ADPKD go beyond direct health care costs."
  • "Moreover, this study provides important insights that may help clinicians target their treatment strategies for improving or delaying disease progression that reduce a rare disease's economic burden."

InMed Receives CTA Approval for Second Phase 1 Clinical Trial with INM-755

Retrieved on: 
Thursday, April 30, 2020
Health, Genodermatoses, Rare diseases, Epidermolysis bullosa, RTT, INMED, Clinical trial

VANCOUVER, April 30, 2020 /PRNewswire/ - InMed Pharmaceuticals Inc. ("InMed" or the "Company") (TSX: IN; OTCQX: IMLFF), a clinical-stage pharmaceutical company developing medications targeting diseases with high unmet medical need and leading the way in the clinical development of cannabinol ("CBN"), today announced it has received regulatory and ethics board approval of its Clinical Trial Application ("CTA") to conduct its second Phase 1 trial with INM-755.

Key Points: 
  • VANCOUVER, April 30, 2020 /PRNewswire/ - InMed Pharmaceuticals Inc. ("InMed" or the "Company") (TSX: IN; OTCQX: IMLFF), a clinical-stage pharmaceutical company developing medications targeting diseases with high unmet medical need and leading the way in the clinical development of cannabinol ("CBN"), today announced it has received regulatory and ethics board approval of its Clinical Trial Application ("CTA") to conduct its second Phase 1 trial with INM-755.
  • The 755-102-HV clinical trial is a randomized, double-blind, vehicle-controlled, Phase 1 study designed to evaluate the safety and tolerability of INM-755 cream applied daily on epidermal wounds in healthy volunteers.
  • INM-755 is a CBN cream intended as a topical therapy to treat epidermolysis bullosa ("EB") and potentially other dermatological diseases.
  • "We are pleased with the successful CTA review process and we look forward to advancing INM-755 into this second Phase 1 trial," commented Eric A. Adams, President and CEO of InMed Pharmaceuticals.

European Medicines Agency Grants Orphan Drug Designation to Amylyx Pharmaceuticals’ AMX0035 for the Treatment of Amyotrophic Lateral Sclerosis (ALS)

Retrieved on: 
Wednesday, April 29, 2020
Biotechnology, General Health, Mental health, Health, Pharmaceutical, Health, Articles, Life sciences, Rare diseases, European Medicines Agency, European Union, Orphan drug, Amyotrophic lateral sclerosis, Medication, Genervon, Leadiant Biosciences, AMX0035, Amylyx Pharmaceuticals

Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has recommended that AMX0035 be designated as an orphan medicinal product for the treatment of ALS.

Key Points: 
  • Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) has recommended that AMX0035 be designated as an orphan medicinal product for the treatment of ALS.
  • The EMA grants Orphan Drug Designation status for products intended for the treatment, prevention or diagnosis of rare, life-threatening or chronically debilitating conditions where the product may represent a significant benefit over existing treatments.
  • ALS patients worldwide are in need of new therapies, said Joshua Cohen, Co-CEO, Chairman, and Co-Founder of Amylyx, Were excited about todays news and look forward to working with the European Medicines Agency on the next steps for AMX0035.
  • Amylyx Pharmaceuticals, Inc. is a pharmaceutical company working on developing a novel therapeutic for Amyotrophic Lateral Sclerosis (ALS), Alzheimers disease and other neurodegenerative diseases.

CytRx Corporation Highlights Updated Arimoclomol Milestone Guidance Provided by Orphazyme A/S in Niemann-Pick Disease

Retrieved on: 
Wednesday, April 29, 2020
Organ systems, Rare diseases, Neurological disorders, Medicine, Lipid storage disorders, Arimoclomol, Piperidines, Inclusion body myositis, Myositis, Niemann, Inclusion bodies, Amyotrophic lateral sclerosis

In addition, in the rest of the world Orphazyme indicated they are finalizing partnering for key countries, including Japan, Canada and Turkey.

Key Points: 
  • In addition, in the rest of the world Orphazyme indicated they are finalizing partnering for key countries, including Japan, Canada and Turkey.
  • CytRx would be eligible to receive future milestone and royalty payments from our arimoclomol agreement.
  • Arimoclomol is in clinical development at Orphazyme for the treatment of Niemann-Pick disease Type C, Gaucher disease, sporadic Inclusion Body Myositis, and amyotrophic lateral sclerosis (ALS).
  • Orphazyme is testing arimoclomol in four indications including amyotrophic lateral sclerosis (ALS), Niemann-Pick disease Type C (NPC), Gaucher disease and sporadic Inclusion Body Myositis (sIBM).CytRx Corporation's website is www.cytrx.com .

uniQure Announces Significant Presence at Upcoming American Society of Gene and Cell Therapy (ASGCT) Virtual Annual Meeting

Retrieved on: 
Tuesday, April 28, 2020
Neurological disorders, Branches of biology, Organ systems, Rare diseases, Autosomal dominant disorders, Trinucleotide repeat disorders, Neurodegenerative disorders, Spinocerebellar ataxia, Fabry disease, Ataxia, Gene therapy, Huntington's disease

and AMSTERDAM, April 28, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 22 data presentations, of which five are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being held May 12-15.

Key Points: 
  • and AMSTERDAM, April 28, 2020 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that 22 data presentations, of which five are oral presentations, will be delivered at the American Society of Gene and Cell Therapy (ASGCT) Virtual 2020 Annual Meeting being held May 12-15.
  • "uniQures significant scientific presence at this years ASGCT Annual Meeting demonstrates the robustness of our research capabilities and our excellence in developing novel technologies and commercial-scale gene therapy manufacturing, stated Sander van Deventer , executive vice president of research and product development at uniQure.
  • We are very pleased to present new preclinical data on our gene therapy candidates for hemophilia A, spinocerebellar ataxia type 3, Fabry disease and data on our advances in technology and manufacturing.
  • We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases.