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A Company in the Fold of Rare Disease
Protein misfolding is an underlying issue for many diseases, including lysosomal storage disorders and some neurodegenerative conditions. […]
Read moreAmbit Partners with Datavant to Drive Innovation in the Rare and Specialty Disease Markets
Rare Daily Staff Ambit, an integrated biopharma solutions firm specializing in the rare and specialty disease markets, […]
Read moreTaysha Discontinues Development of Gene Therapy for Giant Axonal Neuropathy
Rare Daily Staff Taysha Gene Therapies said it is discontinuing the development of its TSHA-120 program for […]
Read moreAI Tool Classifies the Effects of 71 Million Missense Mutations
Rare Daily Staff Google DeepMind has released the AlphaMissense catalogue, which used an AI model to categorize […]
Read moreFDA Grants Fast Track Designation for Kymera’s KT-333 for the Treatment of Two Rare Cancers
Rare Daily Staff The U.S. Food and Drug Administration has granted Fast Track designation to Kymera Therapeutics’ […]
Read moreAIRNA Launches to Develop New Class of RNA Editing Therapeutics for Rare and Common Diseases
Rare Daily Staff AIRNA, a company pioneering RNA editing therapeutics to restore the health of patients with […]
Read moreReCode Therapeutics Raises Additional $50 Million to Advance mRNA Pipeline Closing
Rare Daily Staff ReCode Therapeutics, a clinical-stage genetic medicines company using precision delivery to power the next […]
Read moreBroken String Biosciences Raises $15 Million to Drive Development of Cell and Gene Therapies
Rare Daily Staff U.K.-based Broken String Biosciences, a genomics company building a technology platform to drive the […]
Read moreFDA Approves GSK’s Ojjaara for Rare Blood Cancer
Rare Daily Staff The U.S. Food and Drug Administration has approved GSK’s Ojjaara for the treatment of […]
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