Date |
Product |
Disease |
Phase |
Company |
Therapeutic area |
2018-01-16
|
AVXS-101 (adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene) |
spinal muscular atrophy (SMA) Type 1 |
3 |
Avexis (USA - IL) |
Rare diseases - Genetic diseases - Neuromuscular diseases |
2018-01-16
|
AVXS-101 (adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene) |
spinal muscular atrophy (SMA) Type 1 |
|
Avexis (USA - IL) |
Rare diseases - Genetic diseases - Neuromuscular diseases |
2018-01-15
|
Cx601 (adipose derived allogeneic stem cell therapy) |
perianal fistulas in Crohn's disease patients |
3 |
Tigenix (Belgium) |
Autoimmune diseases - Inflammatory diseases - Digestive diseases |
2018-01-11
|
BI 1467335 (formerly known as PXS-4728A) |
diabetic retinopathy |
2a |
Boehringer Ingelheim (Germany) |
Ophtalmological diseases |
2018-01-10
|
GS030 |
retinitis pigmentosa |
1-2 |
Gensight Biologics (France) |
Rare diseases - Genetic diseases - Ophtalmological diseases |
2018-01-09
|
GNbAC1 |
type 1 diabetes |
2a |
Geneuro (Switzerland) |
Autoimmune diseases - Metabolic diseases |
2018-01-08
|
AR101 |
peanut allergy |
3 |
Aimmune Therapeutics (USA - CA) previously Allergen Research Corporation (ARC) |
Allergic diseases |
2018-01-08
|
Tesomet® (tesofensine) |
Prader-Willi syndrome |
2a |
Saniona (Denmark) |
Rare diseases - Genetic diseases - Metabolic diseases |
2018-01-08
|
MAGE A10c796T - autologous genetically modified T-cells expressing enhanced T cell receptors (TCRs) specific for MAGE A10 |
locally advanced or metastatic (Stage IIIb or IV) non-small cell lung cancer (NSCLC) |
1-2 |
Adaptimmune (UK) |
Cancer - Oncology |
2018-01-08
|
ACE 083 |
facioscapulohumeral muscular dystrophy (FSHD) |
2 |
Acceleron Pharma (USA - CA) |
Rare diseases - Genetic diseases - Neuromuscular diseases |
2018-01-08
|
intepirdine (RVT-101) |
dementia with Lewy bodies (DLB) |
2 |
Axovant Sciences (USA - NY) |
Neurodegenerative diseases |
2018-01-05
|
ISIS 678354 (AKCEA-APOCIII-LRx) |
patients with hypertriglyceridemia and established cardiovascular disease |
2b |
Akcea Therapeutics (USA - CA), a subsidiary of Ionis Pharmaceuticals (USA - CA) |
Cardiovascular diseases |
2018-01-05
|
M281 |
autoimmune diseases |
1 |
Momenta Pharmaceuticals (USA - MA) |
Autoimmune diseases |
2018-01-04
|
BioChaperone® glucagon exenatide |
obesity |
|
Adocia (France) |
Metabolic diseases |
2018-01-04
|
BioChaperone® teduglutide |
short bowel syndrome |
|
Adocia (France) |
Rare diseases - Gastrointestinal diseases |
2018-01-04
|
triheptanoin (UX007) |
long-chain fatty acid oxidation disorder (LC-FAOD) |
2 |
Ultragenyx Pharmaceutical (USA - CA) |
Rare diseases - Cardiovascular diseases |
2018-01-04
|
ABX464 |
ulcerative colitis |
2a |
Abivax (France) |
Autoimmune diseases - Inflammatory diseases - Digestive diseases |
2018-01-03
|
BAL101553 in combination with standard radiation |
newly-diagnosed glioblastoma |
1 |
Basilea Pharmaceutica (Switzerland) Adult Brain Tumor Consortium (ABTC) (USA) |
Cancer - Oncology |
2018-01-03
|
|
solid tumors including advanced or metastatic breast, ovarian, endometrial and urothelial carcinomas |
1 |
Five Prime Therapeutics (USA - CA) |
Cancer - Oncology |
2018-01-03
|
BAL101553 |
advanced solid tumors including glioblastoma |
2a |
Basilea Pharmaceutica (Switzerland) |
Cancer - Oncology |