Lysosomal storage diseases

Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - 2021 Drugs in Development Report - ResearchAndMarkets.com

Retrieved on:

Friday, June 4, 2021

Health, Pharmaceutical, Enzymes, Lysosomal storage diseases, Maltase, Acid alpha-glucosidase, Alpha-glucosidase, Hydrolases, Carbohydrates, Lysosome, Glucosidases, Glucocerebrosidase, GAA, Glucan 1,4-a-glucosidase

The "Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021" drug pipelines has been added to ResearchAndMarkets.com's offering.

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The "Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) - Drugs in Development, 2021" drug pipelines has been added to ResearchAndMarkets.com's offering.
According to the recently published report Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) pipeline Target constitutes close to 19 molecules.
The report outlays comprehensive information on the Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics, complete with analysis by indications, stage of development, mechanism of action (MoA), route of administration (RoA) and molecule type; that are being developed by Companies / Universities.
It also reviews key players involved in Lysosomal Alpha Glucosidase (Acid Maltase or Aglucosidase Alfa or GAA or EC 3.2.1.20) targeted therapeutics development with respective active and dormant or discontinued projects.

Passage Bio to Host Virtual R&D Event on May 17

Retrieved on:

Monday, May 10, 2021

Branches of biology, Granulin, Lysosomal storage diseases, Lysosome, Cell biology

b'PHILADELPHIA, May 10, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc .

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b'PHILADELPHIA, May 10, 2021 (GLOBE NEWSWIRE) -- Passage Bio, Inc .
In approximately 5% to 10% of individuals with FTD, the disease occurs because of mutations in the GRN gene, causing a deficiency of progranulin (PGRN).
The mechanism by which PGRN deficiency results in FTD is uncertain, but increasing evidence points to PGRN\xe2\x80\x99s role in lysosomal function.
Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.\n'

AVROBIO to Present Clinical and Preclinical Data at the ASGCT 24th Annual Meeting

Retrieved on:

Tuesday, April 27, 2021

Biotechnology, Health, Genetics, Pharmaceutical, Clinical trials, Inborn errors of metabolism, Branches of biology, Rare diseases, Lipid storage disorders, Lysosomal storage diseases, Biotechnology, Gene delivery, Gene therapy, Fabry disease, Gaucher's disease, Glycogen storage disease type II

Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease.

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Our ex vivo lentiviral gene therapy pipeline includes clinical programs in Fabry disease, Gaucher disease type 1 and cystinosis, as well as preclinical programs in Hunter syndrome, Gaucher disease type 3 and Pompe disease.
AVROBIO is powered by our industry leading plato\xc2\xae gene therapy platform, our foundation designed to deliver gene therapy worldwide.
Results in preclinical or early-stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval.
AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.\nView source version on businesswire.com: https://www.businesswire.com/news/home/20210427005470/en/\n'

Amicus’ AT-GAA Shows Clinically Meaningful & Significant Improvements in Both Musculoskeletal and Respiratory Measures in Late-Onset Pompe Disease Compared to Standard of Care in Pivotal Phase 3 PROPEL Study

Retrieved on:

Thursday, February 11, 2021

Branches of biology, Clinical medicine, Medicine, Hepatology, Lysosomal storage diseases, Rare diseases, Exercise physiology, Inborn errors of carbohydrate metabolism, Glycogen storage disease type II, Alglucosidase alfa, Pompe, Acid alpha-glucosidase

PROMIS Fatigue: Fatigue as measured by this scale slightly favored AT-GAA treated patients over alglucosidase alfa treated patients.

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PROMIS Fatigue: Fatigue as measured by this scale slightly favored AT-GAA treated patients over alglucosidase alfa treated patients.
Urine Hex-4 is a common biomarker in Pompe disease and is used as an indirect measure of the degree of skeletal glycogen clearance in Pompe patients receiving ERT.
Glycogen is the substrate that accumulates in the lysosomes of muscles of Pompe patients.
We believe that AT-GAA has the potential to quickly become the new standard of care in the treatment of this devastating muscle disease.

Passage Bio Receives FDA Clearance of IND Application for Lead Gene Therapy Candidate PBGM01 for Treatment of Infantile GM1 Gangliosidosis

Retrieved on:

Monday, January 4, 2021

Branches of biology, GLB1, Galactosides, Senescence, Lysosomal storage diseases, Lysosomal storage disease, Lysosome, Neurodegeneration, Galactosidases

FDA clearance of our IND represents a significant milestone that supports the transition of Passage Bio to a clinical-stage company, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.

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FDA clearance of our IND represents a significant milestone that supports the transition of Passage Bio to a clinical-stage company, said Bruce Goldsmith, Ph.D., president and chief executive officer of Passage Bio.
GM1, a rare monogenic lysosomal storage disease, is caused by mutations in the GLB1 gene, which encodes the lysosomal enzyme beta -galactosidase (-gal).
Reduced -gal activity results in the accumulation of toxic levels of GM1 in neurons throughout the brain, causing rapidly progressive neurodegeneration.
I am pleased that Passage Bio has FDA clearance to proceed with this study, said James Wilson, M.D., Ph.D., director of the Gene Therapy Program at the University of Pennsylvania (Penn) and chief scientific advisor of Passage Bio.

Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce Launch of Expanded Access Program in the United States for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease

Retrieved on:

Friday, October 2, 2020

Rare diseases, Lipid storage disorders, Lysosomal storage diseases, Protalix BioTherapeutics, Fabry disease, Clinical medicine, Medical specialties, Fabry, Companies, Priority review

"The National Fabry Disease Foundation and the Fabry community are very excited about the launch of Chiesi's Expanded Access Program for pegunigalsidase alfa for the treatment of Fabry disease," said Jerry Walter, Founder and President, National Fabry Disease Foundation.

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"The National Fabry Disease Foundation and the Fabry community are very excited about the launch of Chiesi's Expanded Access Program for pegunigalsidase alfa for the treatment of Fabry disease," said Jerry Walter, Founder and President, National Fabry Disease Foundation.
When seeking expanded access, treating physicians should consider all possible risks of treatment with pegunigalsidase alfa.
In August 2020, the FDA accepted the pegunigalsidase alfa BLA and granted Priority Review designation for pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease.
Protalix has partnered with Chiesi Global Rare Diseases, both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.

Chiesi Global Rare Diseases and Protalix BioTherapeutics Announce Launch of Expanded Access Program in the United States for Pegunigalsidase Alfa for the Proposed Treatment of Fabry Disease

Retrieved on:

Friday, October 2, 2020

Rare diseases, Lipid storage disorders, Lysosomal storage diseases, Protalix BioTherapeutics, Fabry disease, Clinical medicine, Medical specialties, Fabry, Companies, Priority review

The National Fabry Disease Foundation and the Fabry community are very excited about the launch of Chiesis Expanded Access Program for pegunigalsidase alfa for the treatment of Fabry disease, said Jerry Walter, Founder and President, National Fabry Disease Foundation.

Key Points:

The National Fabry Disease Foundation and the Fabry community are very excited about the launch of Chiesis Expanded Access Program for pegunigalsidase alfa for the treatment of Fabry disease, said Jerry Walter, Founder and President, National Fabry Disease Foundation.
When seeking expanded access, treating physicians should consider all possible risks of treatment with pegunigalsidase alfa.
In August 2020, the FDA accepted the pegunigalsidase alfa BLA and granted Priority Review designation for pegunigalsidase alfa for the proposed treatment of adult patients with Fabry disease.
Protalix has partnered with Chiesi Global Rare Diseases, both in the United States and outside the United States, for the development and commercialization of pegunigalsidase alfa.

CANbridge Pharmaceuticals Receives Chinese Marketing Approval for Hunterase® First Enzyme Replacement Therapy for Hunter Syndrome in China

Retrieved on:

Wednesday, September 9, 2020

This is the first approved treatment in the CANbridge rare disease portfolio and the first ERT treatment in China for Hunter syndrome, where it fills an urgent treatment gap.

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This is the first approved treatment in the CANbridge rare disease portfolio and the first ERT treatment in China for Hunter syndrome, where it fills an urgent treatment gap.
Hunter syndrome (MPS II) is an inherited lysosomal storage disease that is X-linked and occurs primarily in boys.
There is no cure currently, but enzyme replacement therapy (ERT) is unanimously recommended as the standard treatment for MPS II2,3,.
In greater China, where it is a recognized leader in orphan diseases, CANbridge has an exclusive licensing agreement to commercialize Hunterase, an enzyme replacement therapy for the treatment of Hunter syndrome, developed by GC Pharma and marketed in more than 11 countries worldwide.

AVROBIO Announces New Patients Dosed in Gaucher Disease and Cystinosis Clinical Trials

Retrieved on:

Monday, July 6, 2020

The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.

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The company also announced that the second patient has been dosed in the ongoing investigator-sponsored Phase 1/2 clinical trial of AVR-RD-04 for cystinosis.
The first patient dosed is an important milestone for the Gaucher disease community and our AVR-RD-02 program.
AVROBIO is conducting two clinical trials for its AVR-RD-01 investigational gene therapy for Fabry disease.
Our clinical-stage programs include Fabry disease, Gaucher disease and cystinosis and we also are advancing a preclinical program in Pompe disease.

Sanofi to present Phase 3 results of avalglucosidase alfa in patients with late-onset Pompe disease

Retrieved on:

Monday, June 8, 2020

Medicine, Rare diseases, Clinical medicine, Health, Inborn errors of carbohydrate metabolism, Lysosomal storage diseases, Glycogen storage disease type II, Hepatology, Alglucosidase alfa, Pompe, Danon disease, Lysosomal storage disease

Data from the Phase 3 COMET trial would have been presented at the July 2020 ICNMD.

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Data from the Phase 3 COMET trial would have been presented at the July 2020 ICNMD.
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation status to avalglucosidase alfa for the treatment of patients with a confirmed diagnosis of Pompe disease.
Pompe disease is often classified as late-onset Pompe disease (LOPD) or infantile-onset Pompe disease (IOPD).
Avalglucosidase alfa is an investigational ERT for Pompe disease designed to improve the delivery of enzyme to the cells in the muscles, most notably into skeletal muscle.