pharmaphorum

OCTOBER 5, 2022

Sanofi has added to its rare disease pipeline by licensing an antibody-RNA conjugate (ARC) for facioscapulohumeral muscular dystrophy (FSHD), a genetic muscle disorder, from US biotech miRecule.

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RNA Genetics Dermatology Antibody 93

pharmaphorum

JANUARY 10, 2022

Last October, Vertex Pharma cut a similar sized deal with the start-up – co-founded by CRISPR pioneer and Nobel laureate Jennifer Doudna – paying $41 million upfront with $650 million at the back end for in vivo drugs for two unidentified genetic diseases.

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In-Vivo Gene Editing Gene Therapy Gene 52

The Pharma Data

NOVEMBER 29, 2020

Y-mAbs Therapeutics has a target action date of November 30 for its Biologics License Application (BLA) for Danyelza (naxitamab) for patients with relapsed/refractory high-risk neuroblastoma. The drug was developed by researchers at Memorial Sloan Kettering Cancer Center and exclusively licensed to Y-mAbs.

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Genetic Disease Antibody Licensing Licensing 52

Drug Discovery World

JANUARY 30, 2024

Dr Naldini open new doors for cures for otherwise untreatable genetic diseases with the full impact of his work still untapped”, she said. Intellectual property: High patent royalties and licensing fees for key technologies can inflate treatment costs. Fostering open innovation and collaboration could help address this issue.

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The Pharma Data

JANUARY 17, 2021

Based in Seattle, Washington, Sana focuses on in vivo and ex vivo cell engineering platforms to develop therapies for cancer, diabetes, cardiovascular disease, CNS disorders, and genetic diseases. The technology provides a way of producing hypoimmunogenic stem cells that can be differentiated into any cell type.

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In-Vivo Marketing Engineer Genetics 52

XTalks

APRIL 24, 2023

Rare diseases can often be progressive, chronic and fatal. Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Sadly, one-third of children with rare diseases die before their first birthday.

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Gene Therapy Gene Trials Clinical Trials 95

pharmaphorum

SEPTEMBER 17, 2020

Also emerging from the pipeline is Regeneron’s ANGPTL3-targeting antibody evinacumab , which is due for an initial FDA decision in rare genetic diseases that cause elevated cholesterol but also in development for patients with non-inherited forms of high cholesterol who can’t meet treatment targets with current drugs.

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pharmaphorum

OCTOBER 16, 2020

These might be licensed drugs that could hold potential for a patent extension, or drugs which failed efficacy trials for an intended indication. Findacure is a charity that works directly with rare disease patient groups to help them grow and professionalise.

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Clinical Trials Clinical Trials Trials Life Science 90

The Pharma Data

DECEMBER 20, 2020

Athenex and Almirall have a license deal with Almirall having the license to research, develop and commercialize the drug in the U.S. CF is a rare, life-shortening genetic disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. and Europe, including Russia.

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The Pharma Data

NOVEMBER 8, 2020

Bristol Myers Squibb has a target action date of November 16 for the biologics license application (BLA) for lisocabtagene maraleucel (liso-cel), a CD19-directed CAR T cell therapy for adults with r/r large B-cell lymphoma after at least two previous therapies.

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pharmaphorum

OCTOBER 25, 2022

The Japanese firm has agreed to make a $50 million investment in Dallas-based Taysha in exchange for a 15% stake in the company, plus exclusive options to license two clinical-stage, single-gene therapies for rare genetic diseases.

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Gene Therapy Gene Genetic Disease Genetics 77

pharmaphorum

DECEMBER 8, 2021

If approved, the drug will be sold by Neuren Pharma, which licensed North American rights to the drug in 2018 and is also developing another drug for Rett blarcamesine, in collaboration with Anavex Life Sciences. Results from a phase 3 trial of blarcamesine are due next year.

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Marketing Drugs Life Science Trials 57

Delveinsight

AUGUST 17, 2021

Food and Drug Administration, which could have entered the ultra-rare genetic disease market. The rarity of disease – 1.36 Seagen will pay USD 200 million upfront to RemeGen and also secure an exclusive worldwide license agreement of upto USD 2.4 person/million around the globe.

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FDA Approval Medicine Vaccination Vaccine 98

The Pharma Data

DECEMBER 8, 2020

The impact of CNS diseases extends beyond patients—to their families and society as well.” The assumption is SciNeuro will start with Parkinson’s disease and pain in addition to rare genetic diseases, after scaling up staffing. .

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RNA Antibody Life Science Protein 52

The Pharma Data

NOVEMBER 11, 2020

Food and Drug Administration (FDA) for INZ-701 for the treatment of ENPP1 deficiency – The FDA grants rare pediatric disease designation to drugs for serious and life-threatening diseases in which the serious or life-threatening manifestations primarily affect children aged from birth through 18 years and affect fewer than 200,000 people in the U.S.

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Clinical Trials Clinical Trials Trials Development 40

XTalks

DECEMBER 29, 2022

Vertex is planning to submit a Biologics License Application (BLA) by the end of the first quarter of 2023 and the company says it is also on track to submit exa-cel to UK and European Union (EU) regulators by the end of the year. As such, most gene therapies in late-stage clinical trials are in oncology or rare disease indications.

Life Science 52

Life Science Gene Therapy Gene Manufacturing 52

pharmaphorum

OCTOBER 26, 2020

The company’s most advanced programmes are in Pompe disease – a rare genetic disease causing buildup of a sugar molecule inside cells – Parkinson’s disease and congestive heart failure.

Gene Therapy 57

Gene Therapy Gene Genetic Disease Genetics 57

XTalks

SEPTEMBER 17, 2020

Symptoms and Etiology: Characterized by progressive muscle weakness and atrophy, Duchenne muscular dystrophy (DMD) is an X-linked genetic condition that primarily affects males. Patients with this disease have a very high level of LDL-C, which increases their risk of coronary artery disease, including heart attack, stroke and atherosclerosis.

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Trials Gene Hormones Clinical Trials 98

The Pharma Data

NOVEMBER 2, 2020

Europe, Latin America, the Middle East, and most of Asia Pacific, there are currently no licensed medicines for achondroplasia. BioMarin is a global biotechnology company that develops and commercializes innovative therapies for serious and life-threatening rare and ultra-rare genetic diseases. In the U.S., About BioMarin.

Drugs 40

Drugs Containment Trials Marketing 40

Drug Discovery World

MARCH 6, 2024

The development of effective new disease-modifying treatments in neurodegenerative and neuropsychiatric disorders has been hindered by their inherent genetic complexity, environmental influences, and clinical variability. This is comparatively easier to accomplish in these diseases because they are relatively monogenic.

Genome 64

Genome Genomics Clinical Trials Clinical Trials 64

The Pharma Data

AUGUST 3, 2021

The use of a sham procedure in STEER is included to provide a comparison group for an unbiased collection and assessment of efficacy, safety and tolerability of OAV-101 IT for this older population where the disease progression is slower. Spinal Muscular Atrophy (SMA) ?

Clinical Trials 52

Clinical Trials Clinical Trials Gene Therapy Trials 52

The Pharma Data

OCTOBER 14, 2020

As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases.

Protein 52

Protein RNA Production Genetics 52

The Pharma Data

OCTOBER 15, 2020

Sabbagh will be responsible for expanding Inozyme’s proprietary pipeline by identifying and developing new therapeutics for monogenic and non-genetic diseases of abnormal mineralization. Prior to joining Inozyme, Sabbagh served as the head of rare renal and musculoskeletal diseases research at Sanofi.

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In-Vivo Business Development Manufacturing Development 52

The Pharma Data

OCTOBER 28, 2020

GeneTx licensed the rights to antisense technology intellectual property from the Texas A&M University System in December 2017. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultra-rare genetic diseases. About Ultragenyx.

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Protein Production Development Drugs 40

Drug Discovery World

FEBRUARY 22, 2024

For diseases as serious and debilitating for patients and their families as leukodystrophies, learning about such advances in knowledge carries a great deal of hope, which IRCM warmly welcomes,” adds Dr Jean-François Côté, the IRCM’s President and Scientific Director.

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Drugs Gene Clinical Trials Clinical Trials 59