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FDA planning new guidance, hires in cell and gene therapies for 2019

by Conor Hale |Associated Press, January 15, 2019


The FDA plans to hire at least 50 new clinical reviewers tasked with assessing cell and gene therapies to prepare for what the agency describes as a surge of cutting-edge products currently entering early development.

From the end of next year going forward, the agency expects to receive at least 200 investigational new drug (IND) applications annually from companies looking to begin clinical testing. The FDA predicts those will translate into 10 to 20 cell and gene therapy approvals each year by 2025.

Those 200 annual IND applications will build upon the more than 800 active cell-based or directly administered gene therapies that the agency currently has on file, according to a statement from FDA Commissioner Scott Gottlieb, M.D., and CBER Director Peter Marks, M.D., Ph.D.

“The activity reflects a turning point in the development of these technologies and their application to human health,” they wrote. “It’s similar to the period marking an acceleration in the development of antibody drugs in the late 1990s, and the mainstreaming of monoclonal antibodies as the backbone of modern treatment regimens.”

Gottlieb and Marks drew parallels between the innovative platforms that allowed engineered antibodies to be humanized, unlocking their potential by allowing them to slip past the body’s immune system, with the adoption of adeno-associated virus vectors for the delivery of gene therapies.



ARM released State of the Gene and Cell Therapy Industry report

San Francisco, January 7,  2019

During the JP Mprgan Annual Healthcare Conference the 2019 State of  the Cell and gene Therapy Report was released by The Alliance of Regenerative Medicine (ARM).

Here are some highlights:

Global financing in                 2018

  • Gene therapy                $ 9.7B           Up 64%

  • Cell therapy                   $ 7,6 B         Up 64% 

  • Tissue Engineering       936.9M       Up 258%     

Gene Therapy Clinical Studies end of 2018:



Phase 1          120                                                             

Phase 2          210                                                              

Phase 3            32                                                                        



READ THE FULL REPORT FROM ARM                             

Neurocrine pays Voyager $165M for neurological gene therapies

January 29, 2019

Biosciences is set to pay Voyager Therapeutics $165 million upfront for the rights to a clutch of gene therapies. The deal sees Neurocrine commit to $1.7 billion in milestones in return for Parkinson’s disease program VY-AADC and other assets.

VY-AADC is the most advanced of the four assets covered by the agreement. The gene therapy, which is designed to equip patients to convert levodopa into dopamine, is currently being tested in phase 2. Once the phase 2 data are in, Voyager has the option to co-commercialize VY-AADC and split the costs or profits 50/50 or cede full global rights to Neurocrine in return for milestones and royalties.

The VY-AADC program is tied to $170 million in milestones and a royalty rate that could rise to 30% in certain circumstances. Like the three other programs covered by the deal, VY-AADC is also tied to up to $275 million in commercial milestones