Spark Therapeutics (ONCE), focuses on the development of gene therapy products for patients suffering from debilitating genetic diseases.
ONCE’s products include voretigene neparvovec, which is in Phase III clinical trial for the treatment of genetic blinding conditions called inherited retinal diseases caused by non-sex-linked, autosomal recessive, or biallelic mutations in the RPE65 gene; and SPK-CHM that is in Phase I/II clinical trial for the treatment of choroideremia.
ONCE's products also comprise SPK-7001 that is in Phase 1/2 trial for choroideremia; SPK-9001, which is in Phase 1/2 trial for hemophilia B; and SPK-8011 that is in Phase 1/2 trial hemophilia A. In addition, its product candidates and development programs include SPK-FVIII program to treat hemophilia A; SPK-TPP1 program for the treatment of a form of Batten disease and Huntington's disease; RhoNova for the treatment of rhodopsin-linked autosomal dominant retinitis pigmentosa; and SPK-LHON for treating Leber hereditary optic neuropathy, as well as preclinical programs in development for the treatment of rare genetic blinding conditions, hematologic disorders, and other neurodegenerative diseases.
ONCE has collaboration agreement with Pfizer, Inc. for the development and commercialization of SPK-FIX product candidates in its gene therapy program for the treatment of hemophilia B.
Spark Therapeutics, Inc. was founded in 2013 and is headquartered in Philadelphia, Pennsylvania.
March 24, 2016
Lower open expected; RegMed sector, another nudge toward contracting valuations
March 23, 2016
RegMed’s close: the sector downdrafts, I can’t resist - a told you so!
March 23, 2016
Flat to lower open expected; RegMed sector, is it better to profit from a sale or be sorry about having stayed too long?
March 22, 2016
RegMed’s close: the process of investing – speculation
March 22, 2016
uniQure (QURE) Pre-clinical data supports Huntington's disease gene therapy program
March 21, 2016
RegMed’s close: low to average volatility pushes upside momentum
March 21, 2016
Slightly higher open expected; RegMed sector, do you need a few stock trading clues?
March 19, 2016
RegMed’s close: options expiration seemed to be a non-event as volatility swung lower
March 18, 2016
Higher open expected; RegMed sector, quadruple witching, it’s still all about treading carefully
March 17, 2016
RegMed’s close: another dip on low volume and a reset to a positive close
35 companies, 1 interpreter!
Insight, foresight and recommendation
Spark Therapeutics (ONCE) -- Opened 1/18 at $53.31, slipped to $50.56 (1/5) ending the month at $56.05 and started February at $56.30 and has been slippin, dipping and retesting lows such as 2/20's $50.23 a believer. ONCE has built a fully integrated company dedicated to discovering, developing and delivering one-time treatments that provide long-lasting, transformative outcomes to patients, families, society and the health care system. In 2018, ONCE is focused on launching LUXTURNA in the U.S. and securing marketing authorization in the EU, advancing its global development program for SPK-8011 in hemophilia A and also continuing to progress its pipeline of other investigational gene therapies. Recent: entered into an amendment to the license agreement for SPK-9001 with Pfizer (PFE), in 11/17; including an initial $10 million cash payment and up to an additional $15 million in potential milestone payments upon completion of certain transitional activities in mid-2018 and also ntered into a supply agreement with PFE in February 2018 to begin production this quarter for one batch of drug substance expected to be used for Phase 3 development; Spark received $7 million up front and will receive up to $7 million upon delivery. Dips are always due to market conditions but, with $540.2 M in cash excludes the $105 million received from Novartis in 1/18 and is eligible to receive an additional $25 million upon approval by European Medicines Agency (EMA) and total of $40 million in aggregate additional milestones on initial sales in multiple ex-U.S. markets
buyMy motto … never leave an investor uninformed! I say today what others won’t, so you can do what others can’t.
My name is Henry McCusker; I endorse these opinions under my own name and with a subscription based membership.
Do you have an opinion you would like to share with us? We would like to hear from you.
Please e-mail us at hwm@regmedinvestors.com.
Editor and Publisher ... Henry McCusker enters his tenth (10th) year at RegMed Investors
© 2026 RegMed Investors (regmedinvestors.com), all rights are reserved.
RegMed Investors (RMi) or Regmedinvestors.com content is the intellectual property of Henry McCusker. Any copying, republication or redistribution of RMI’s content, including by framing or similar means, is expressly prohibited without the prior written consent of Scimitar; who also shall not be liable for any errors or delays in content, or for any actions taken in reliance thereon. For additional information on other Scimitar media services or reposting please email hwm@regmedinvestors.com.
The information included above as well as individual companies and/or securities mentioned should be construed as guidance in RMi reviews.
The facts stated and the opinions given in this publication are based on data and information considered to be reliable and have been carefully worked into my qualitative analyses and prognoses. However, no guarantee can be given as to their fairness, accuracy or completeness.
Henry McCusker, the editor and publisher of RegMed Investors does not hold or have positions in securities referred to in this publication.