Company Overview and News

 
Gene Therapy, Gene Editing and Immuno-Oncology Creating Thousands of New Job Opportunities

14h biospace
As gene therapy gains traction, it is expected to create a number of job opportunities. For example, the UK government projects gene and cell therapy in Britain alone will create 18,000 new jobs by 2030. The U.S. Bureau of Labor Statistics predicts a 7 percent increase in jobs for biomedical engineers and a 13 percent increase in medical scientists.
GILD BMRN ONCE

 
Using Lab-Grown Retinas, Researchers Take Steps Toward Curing Colorblindness

2018-10-12 biospace
Researchers at Johns Hopkins University, the University of California, San Diego (UCSD), and the National Institute of Mental Health grew retinas in Petri dishes, and discovered fundamental factors involved in the development of vision and color vision. Their research was published in the journal Science.
ONCE

6
Novartis Presents Positive Data on Sickle Cell Disease Drug

2018-10-10 zacks - 1
Novartis AG (NVS - Free Report) announced positive results from a post hoc analysis of the phase II study, SUSTAIN, on pipeline candidate, crizanlizumab.
CNP NVS GSK GSK ONCE

 
Spark Therapeutics Announces New Preclinical Data for Pompe Disease Gene Therapy Candidate

2018-10-08 globenewswire
PHILADELPHIA, Oct. 08, 2018 (GLOBE NEWSWIRE) -- Spark Therapeutics (NASDAQ:ONCE), a fully integrated, commercial gene therapy company dedicated to challenging the inevitability of genetic disease, today announced new data for SPK-3006, an investigational liver-directed adeno-associated viral (AAV) gene therapy for Pompe disease. The data from the Investigational New Drug (IND)-enabling studies were presented Saturday, Oct.
ONCE

 
With Hemlibra, Roche seeks to break into tight hemophilia circle

2018-10-03 channelnewsasia
Swiss drugmaker Roche is breaking into hemophilia A treatment, a US$10 billion global market dominated by rivals who have cultivated close ties to sufferers of the genetic bleeding disorder.
ONCE

1
Survey Says: Top 5 Trends in the Life Sciences Industry

2018-09-26 biospace - 1
Recently, BioSpace posted a question on its website, under the heading, “We Want to Hear from You! Top Life Sciences Trends.” And don’t stop! We still want to hear from you!
HGRL BMRN ONCE

 
Spark Therapeutics gibt zustimmende Bewertung des Ausschusses für Humanarzneimittel für die einmalig verabreichte Gentherapie LUXTURNA® (Voretigen Neparvovec) in der Europäischen Union bekannt

2018-09-25 globenewswire
LUXTURNA wäre die erste Gentherapie für eine genetische Erkrankung, die sowohl in den USA als auch in der EU zugelassen ist
ONCE

 
Spark Therapeutics Annuncia il Parere positivo del CHMP per la Terapia Genica una tantum LUXTURNA® (voretigene neparvovec) nell’Unione europea

2018-09-25 globenewswire
LUXTURNA diventerebbe la prima terapia genica per una malattia genetica approvata sia negli USA sia nella UE LUXTURNA sarebbe la prima e unica terapia genica approvata nell’UE per il trattamento di quei pazienti che presentano un numero sufficiente di cellule retiniche vitali e che hanno perso la vista a causa di distrofia retinica ereditaria associata a mutazioni bialleliche comprovate del gene RPE65, una condizione che porta alla cecità totale nella maggioranza dei pazienti
ONCE

5
Novartis Gets Positive CHMP Opinion for Drugs, Biosimilar

2018-09-24 zacks
Novartis AG (NVS - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of Gilenya for the treatment of children and adolescents aged 10 to 17 years with relapsing remitting forms of multiple sclerosis (RRMS).
REGN RDN NVS GSK GSK ONCE

 
Spark's Luxturna blindness therapy gets European panel nod

2018-09-21 reuters
ZURICH, Sept 21 (Reuters) - A European Medicines Agency panel recommended approval of Spark Therapeutics’ gene therapy for blindness, a move that also boosts Swiss drugmaker Novartis that bought the rights to one of the world’s costliest treatments outside the United States.
ONCE

1
Novartis announces positive CHMP opinion for one-time gene therapy Luxturna® to treat children and adults with rare inherited retinal disease

2018-09-21 globenewswire
Basel, September 21, 2018 - Novartis today announced that the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the approval of voretigene neparvovec, a one-time gene therapy for the treatment of patients with vision loss due to a genetic mutation in both copies of the RPE65 gene. Luxturna was developed and is marketed in the US by Spark Therapeutics. If approved, voretigene neparvovec will be commercialized by Novartis in markets outside the U.
NVS ONCE

 
Spark Therapeutics Announces Positive CHMP Opinion for One-time Gene Therapy LUXTURNA® (voretigene neparvovec) in the European Union

2018-09-21 globenewswire
LUXTURNA would be first gene therapy for a genetic disease approved in both U.S. and EU
ONCE

 
Krystal Biotech: Buy The Dip

2018-09-21 seekingalpha
A private placement was a solid indication of institutional interest and granting of Rare Pediatric Disease Designation could lead to receipt of a valuable voucher later on.
JAZZ KRYS ONCE

 
Regenxbio (Part IV): Elucidating The Prospects Of The AMD Franchise

2018-09-17 seekingalpha
Regenxbio is a stellar gene therapy innovator that is delivering hope to patients while handsomely rewarding investors.
ONCE RGNX

22
Sangamo (Part V): Assessing The Prospects Of SB-913

2018-09-11 seekingalpha - 2
Cicero learned man that he was, believed in self-improvement so long as his breath lasts. He commends Socrates for learning to play the fiddle late in life and another Roman for mastering Greek when old. - Charlie Munger
SHPG GILD SGMO KITE PFE BIVV ONCE RGNX

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Silicon Investor Message Boards

This table lists all message boards related to ONCE / Spark Therapeutics, Inc. on message board site Silicon Investor.

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CUSIP: 84652J103