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J.P. Morgan Roundtable: The Regulatory Role In Accelerating Complex Therapies

 
• By Mandy Jackson and Mike Ward

Part 3: Scrip spoke with companies working on cell and gene therapies and novel approaches to neurological diseases during the J.P. Morgan Healthcare Conference about how regulators are supporting novel modalities, which in turn improves investor and partner sentiment.

JPM Roundtable 3

Regulators around the world are helping companies with innovative regenerative medicine platforms and other novel technologies get their medicines to patients as quickly as possible – and prospective investors and partners are taking notice.

Roundtable Participants

BlueRock Therapeutics VP of Corporate Development and Strategy Eric Soller. BlueRock uses induced pluripotent stem cells (iPSCs) to develop engineered cells in the areas of neurology, cardiology and autoimmunity. Lead programs include a dopaminergic neuron for Parkinson's disease moving into the clinic this year, a cardiomyocyte for congestive heart failure, and a macrophage to treat autoimmune diseases. (Also see "Early-Stage Cell And Gene Therapy Progress: Updates From Casebia, BlueRock, Athersys, Cynata" - Scrip, 11 October, 2018.)

United Neuroscience (UNS) CEO Mei Mei Hu. UNS is leveraging vaccine technology commercialized in more than 4bn doses – primarily in animal health – to develop therapeutic vaccines. SC123854 The company recently reported positive Phase IIa data for its lead candidate targeting amyloid beta in Alzheimer's disease. (Also see "United Neuroscience To Move Forward Alzheimer's Vaccine After Promising Phase IIa" - Scrip, 17 January, 2019.) 

Athersys Chairman and CEO Gil Van Bokkelen. Athersys is developing MultiStem from adult stem cells derived from donated bone marrow and other tissues. It is in Phase III with a regenerative medicine advanced therapies (RMAT) designation from the FDA for ischemic stroke; it's in mid-stage studies for heart attack and acute respiratory distress syndrome. Healios KK holds a license for MultiStem in Japan.

PTC Therapeutics Inc. Chief Scientific Officer Mark Pykett. PTC's portfolio includes the commercial Duchenne muscular dystrophy (DMD) drugs – Emflaza (deflazacort) in the US and Translarna (ataluren) outside the US (estimated combined sales of $280m-$305m in 2019) – and various rare disease and oncology candidates. PTC got into gene therapy by acquiring Agilis Biotherapeutics LLC last year; Pykett was president and CEO of Agilis. Lead gene therapy candidate GT-AADC for aromatic L-amino acid decarboxylase (AADC) deficiency will be submitted for US FDA approval in 2019.

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