Action plan 2011-2013

6 - TREAT-NMD Advisory Committee for TherapeuticsBiobanks


1 -

TREAT-NMD Advisory Committee for Therapeutics (TACT)

Arrange and conduct a 6 monthly review meeting and produce reports to applicants based on recommendations of the expert reviewers. Disseminate non-confidential output from TACT, engage with funders, solicit and follow up on potential applicants.

Achievements 2011-2013

TACT review meetings

3th TACT meeting: January 2011, Charlotte, North Carolina, USA

  1. Dr Higgins, Paratek Pharmaceuticals: Tetracycline Derivatives as SMN2 Splicing Modifiers for the Treatment of SMA.
  2. Dr Sachs, Rose Pharmaceuticals. GsMTx4: Therapy for Muscular Dystrophy by Inhibition of Mechanosensitive Ion Channels.

4th TACT meeting: October 2011, Lisbon, Portugal

  1. Marc B Blaustein, Halo Therapeutics LLC: A randomised, double-blind, placebo-controlled, multiple-dose, dose-escalation study to evaluate the safety, tolerability, pharmacokinetic, and pharmacodynamic effects of HT-100 in patients with Duchenne muscular dystrophy.
  2. Fabrizio Dolfi, NicOx SA: A 6-month multicenter, randomised, double-blind, placebo-controlled, Phase IIa proof of principle study of naproxcinod (HCT 3012) 750 mg bid in patients with Becker Muscular Dystrophy.
  3. Chris N Airriess, California Stem Cell: Human Embryonic Stem Cell Derived Motor Neuron Progenitors for the Treatment of Motor Neuron Disease.

5th TACT meeting: April 2012, Arlington, Virginia, USA

  1. Fred Marin, GMP-Orphan SAS: A new drug formulation development program of sodium phenylbutyrate in SMA patients.
  2. Urs Ruegg & Olivier Dorchies, University of Geneva: Proposal for clinical investigation of tamoxifen in DMD boys.
  3. Joel Braunstein, Tivorsan Pharmaceuticals: Recombinant Biglycan for Treatment of Duchenne and Becker Muscular Dystrophy.
  4. Paolo Bettica, Italfarmaco SpA: A two part study to assess safety and tolerability, pharmacokinetics, effects on histology and on different clinical parameters of Givinostat in ambulant children with Duchenne Muscular Dystrophy.

6th TACT meeting: October 2012, Prague, Czech Republic

  1. Dariusz C Gorecki, University of Portsmouth: P2X7 purinoceptor as a target for pharmacotherapy of Duchenne Muscular Dystrophy.
  2. Patricio Sepulveda Myostin Therapeutics Pty Ltd: Novel myostatin antagonist peptides to enhance muscle function.
  3. Erica Reeves ReveraGen BioPharma Inc: VBP15 for the treatment of DMD.
  4. Joanne M. Donovan, Catabasis Pharmaceuticals Inc: CAT-1004, a novel anti-inflammatory agent for treatment of Duchenne Muscular Dystrophy.

7th TACT meeting: April 2013, Baltimore, Maryland, USA

  1. Jon Tinsley, Summit PLC: Utrophin modulator for the treatment of Duchenne muscular dystrophy.
  2. Peter Flynn, Fate Therapeutics: Wnt7a Analog Protein Therapeutic for the Treatment of Muscular Dystrophy.

8th TACT meeting: November 2013, Newcastle-upon-Tyne, UK

  1. Jens Schmidt, University Medical Centre Göttingen: Blockade of nitric oxide-related cell stress as potential treatment for inclusion body myositis
  2. Denis Guttridge, Ohio State University: NBD Therapy for Duchenne Muscular Dystrophy
  3. Carl Morris, Rare Disease Research Unit, Pfizer Inc: Anti-GDF8 antibody for treatment of Duchenne Muscular Dystrophy
Communication and dissemination of TACT output

TACT mentioned in Wall Street Journal: December 27, 2011
TACT model presented at Faster Cures conference in New York, 28-30 November 2012

12 Apr 2017