From the Editor April 2016

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Included with this issue is our first of two supplements this year focused on cell therapies. It has been fascinating to watch this journey toward maturation of advanced therapeutics. Some early assumptions that commonalities with protein production would resolve manufacturing problems are being tempered now that we know more about the unique demands of cell and gene therapy development. Still, the future looks brighter for these new modalities.

As Invetech’s Richard Grant (global VP, cell therapy) noted in his plenary address at the January Cell and Gene Therapy World conference in Washington, DC, advanced therapies are poised to revolutionize healthcare by 2025. The past year has seen progress toward removing three main obstacles to commercial success: solving technical challenges, driving down treatment costs, and aligning global regulatory approaches.

One challenge emphasized in this month’s supplement is reduction of manual intervention. It is clear now that scalable commercial solutions will never be manual processes. Contamination risk must be strictly controlled through closed, automated processes, and costs must be controlled too. I was startled to hear that the US cost of one therapeutic administration could equal 25% of the total healthcare costs of some countries! Convergence of regulations around the world (especially in the diagnostics side) is increasing harmonization overall. Grant’s key point was that if regenerative medicines move away from being a science experiment to being a business, then they can change the world.

Another way to view the progress of this industry segment is in the list of “Top 10 Cell and Gene Therapy Events of 2015,” presented at the same conference by Ed Field (president of BioRestorative Therapies and E BioConsulting LLC). Based on his own survey, he counted down the following key milestones over the past year (shared here with his permission):

10: AMAG Pharma buys Cord Blood Registry for US$700 million.

9: Partnerships — BMS and uniCure; Biogen and AGTC

8: Valeant buys Dendreon out of bankruptcy for $495 million.

7: CRISPR — the hottest new technology (patent interference case filed with the US PTO)

6: Massive rounds of funding closed (Reneuron for $105 million; Humacyte for $150 million)

5: Phase 3 trial successes reported for Spark Therapeutics and Tigenix

4: Fujifilm acquires Cellular Dynamics for $307 million, bringing massive resources into the field

3: Positive clinical data for Cellectis’s allogeneic model (codeveloped with Pfizer)

2: Astellas bids to acquire Ocata for $379 million (finally accepted in February 2016, another sign of Big Pharma’s increasing involvement in regenerative medicine)

1: Japanese approval and reimbursement for Temcell and HeartSheet.

Many of those advances are touched on in our supplement this month; others will be discussed in our second cell therapy issue this autumn. Once you have a chance to read this set of articles, I look forward to hearing what you would especially like to see us follow up on. Celebrating progress is necessary; helping drive it is BPI’s editorial mission.