Dyne’s myotonic dystrophy antisense therapy reduces gene expressionDyne Therapeutics Inc. (NASDAQ:DYN) reported data from a new mouse model of severe disease showing its myotonic dystrophy type 1 (DM1) antisense oligonucleotide therapy reduced nuclear DMPK RNA levels...

DISEASE CATEGORY: Cancer INDICATION: Brain cancer Inhibiting epigenetic regulator BMI1 alone or in combination with BCL-2 inhibition could treat diffuse intrinsic pontine glioma (DIPG), an aggressive childhood brain cancer. A screen in two human DIPG...

This year’s ASH meeting provides an opportunity to take stock of clinical progress in hot target, modality and disease areas: CD47 inhibitors, allogeneic CAR T cells and sickle cell disease. Abstracts...

The move toward sickle cell disease therapies that address the pathology’s root causes is reflected in promising early-stage readouts at ASH, which include both disease event rates and changes in red blood cell biology. The...

As targeted protein degradation grows up, the field is going after harder targets and new mechanisms of action. Over the last year, a series of Phase I/II readouts from front-runner Arvinas Inc. (NASDAQ:ARVN),...

Genesis and Genentech collaborate to identify drug candidates using AIStanford spinout and AI platform company Genesis Therapeutics Inc. entered into a collaboration agreement with the Genentech Inc. unit of Roche (SIX:ROG; OTCQX:RHHBY) to identify drug candidates...

After the failure of its lead anti-aging program in osteoarthritis, Unity believes more objective clinical endpoints and robust preclinical data for its next-in-line ophthalmology candidate mean that program won’t suffer the same fate in the...

By enriching its coffers with a further $2.1 billion from existing shareholders, BeiGene gains a formidable cash position as it brings to market a handful of products in the U.S. and China and advances a...

Venclexta combo shows survival benefit in AML Partners Roche (SIX:ROG; OTCQX:RHHBY) and AbbVie Inc. (NYSE:ABBV) said Venclexta venetoclax plus azacitidine significantly improved overall survival and composite complete remission rate among acute myelogenous leukemia (AML) patients...

Priority Review for MorphoSys follows Incyte deal FDA accepted and granted Priority Review to a BLA for tafasitamab from MorphoSys AG (NASDAQ:MOR; Xetra:MOR) to treat relapsed or refractory diffuse large B cell lymphoma in combination...