Neuroscience has quietly become a hot space for startups. Across a range of neurologic conditions, contrarian investors have been fueling entrepreneurs to discover and develop novel therapeutic strategies.
AVROBIO, Inc., a clinical-stage biotechnology company developing transformative, life-changing gene therapies for rare diseases, today announced the expansion of its pipeline to Pompe disease. This pre-clinical program becomes AVROBIO’s third gene therapy for Lysosomal Storage Disorders (LSDs), following on the heels of the Company’s Phase 1 Fabry program and pre-Phase 1/2 Gaucher program. AVROBIO’s gene therapy for Pompe disease utilizes a proprietary lysosomal targeting sequence to deliver high levels of enzyme to lysosomes.
After quietly scrapping a $485 million buyout option held by Biogen, Cambridge, MA-based Rodin Therapeutics has lined up a $27 million round to get its HDAC inhibitor in the clinic with a plan for taking their first clinical stab at Alzheimer’s — one of the toughest targets in biotech.
Last month, Bloomberg bemoaned the dearth of biotech M&A, and pundits echoed the sentiment (here, here), citing concerns about tax rates and drug pricing.
Bicycle to receive an upfront payment of $10 million, with potential for future milestones.
Bioverativ Inc. (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialisation of innovative therapies for haemophilia and other rare blood disorders, and Bicycle Therapeutics Ltd., a biotechnology company pioneering a new class of therapeutics based on its proprietary bicyclic peptide (Bicycle®) product platform, today announced a research collaboration focused on the discovery, development and commercialisation of innovative therapies for haemophilia and sickle cell disease.