Gene therapies provide life-changing outcomes for patients and some have the potential of being curative.  The market for viral vector-based gene therapies has advanced in recent years with the first approvals occurring in 2017 and 2019 for Roche’s Luxturna® and Novartis’ Zolgensma®, respectively.  In 2019, the US Food and Drug Administration (FDA) predicted a significant increase in the number of cell and gene therapy Investigational New Drug (IND) applications and approvals between 2020 and 2025¹.  As innovator companies plan the clinical path for their gene therapy, strategies for manufacturing and clinical supply should be developed early to help avoid delays in later Phase studies or regulatory review.

Takeda has teamed up with Anima Biotech to discover mRNA translation modulators. The Japanese drugmaker is set to pay up to $120 million in upfront fees and preclinical research milestones to work with Anima on the treatments for genetically defined neurological diseases.

The hype around antibody-drug conjugates (ADCs) has been growing in recent years after big bucks deals and approvals from the likes of AstraZeneca/Daiichi and Immunomedics putting a spotlight on the approach.

The founders of microbiome-testing startup uBiome have been charged with defrauding investors out of $60 million by convincing them it was a successful business before being forced to shutter operations, declare bankruptcy and flush all its assets in late 2019, according to the Securities and Exchange Commission (SEC).

Aktis Oncology has raised a $72 million series A round to develop radiopharmaceutical treatments for solid tumors. The MPM Capital-founded biotech received investment from Novartis and Bristol Myers Squibb.

Mark Rus, formerly Shire’s group vice president of neuroscience, is taking the helm at Delix Therapeutics, a biotech developing drugs that target neuroplasticity to treat central nervous system disorders.

Merck is adding another partner in neurodegenerative disease to the roster: Amathus Therapeutics, a biotech taking aim at molecular chaperones, a group of proteins that guide the folding of other proteins.

The private equity firm Accelmed Partners has closed its second fund, with plans to invest $400 million in the development of commercial-stage health technology companies.

Mike Cloonan is set to step down as chief operating officer of Sage Therapeutics. Cloonan is leaving to pursue a CEO position in the biopharma industry shortly after being overlooked for the top job at Sage.

Bluebird bio’s chief medical officer David Davidson, M.D., is hitting the exit, though remaining as a consultant, as the biotech looks to navigate through its gene therapy issues.