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MiNA and BioMarin partner to speed development of RNAa therapies

Pharmaceutical Technology

MiNA Therapeutics has entered into a research collaboration and option licensing agreement with BioMarin Pharmaceutical to speed up the development of therapeutic ribonucleic acid activation (RNAa) candidates to treat rare genetic diseases. The option licensing agreement is based on early-stage clinical results.

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Birinapant licensing; AvantGen, IGM pairs up for anti-SARS-CoV-2 antibodies; BeiGene, Novartis to co-dvelop Tislelizumab; Valo raises USD190 M; Bluebird Bio Spins-off

Delveinsight

Medivir, IGM Biosciences enters into an exclusive licensing agreement for Birinapant. Medivir AB has entered into an exclusive licensing agreement with IGM Biosciences to receive global, exclusive development rights for Birinapant. AvantGen Enters into a Licensing Agreement for its Anti-SARS-CoV-2 Antibodies with IGM Biosciences.

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Bayer trumpets $1bn CRISPR deal with Mammoth Bio

pharmaphorum

Bayer has bolstered its cell and gene therapy platform by securing access to a CRISPR-based gene-editing platform developed by US biotech Mammoth Biosciences. CRISPR drugs can be used to modify the expression of disease-associated proteins in the body, for example, by correcting a mutation in a specific gene.

In-Vivo 52
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Top 10 Fastest Growing Biotech Companies in 2023

XTalks

The year 2022 has proven to be a momentous period for Fulgent Genetics, marked by significant expansion of its product portfolio across various medical conditions. Xoma’s unique approach involves acquiring future economic interests tied to pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotech companies.

Genetics 111
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FDA Action Alert: Urovant, Athenex, scPharmaceuticals and Vertex

The Pharma Data

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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FDA Action Alert: Supernus, Adamis, Alkermes, Amgen, BMS and Eiger

The Pharma Data

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.

Trials 52
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Surrogate Endpoints: How to Choose the Best One for Your Rare Disease Trial

XTalks

Inherited mutations in the SLC3A1 and SLC7A9 genes cause cystine to be abnormally transported within the kidney leading to cystinuria. Dietary changes, including restriction of salt and animal protein, are also recommended. Individuals with this disease are deficient in ?-galactosidase Mutations in the gene encoding ?-Gal

Trials 98