

# Event Details

- **Event Name**: The Development of Spinraza, a Disease-Modifying Therapy for SMA
- **Event Start and End Date**: Tue, 03 Feb, 2026 at 05:00 pm – Tue, 03 Feb, 2026 at 06:30 pm
- **Event Description**: Join us as we dive into the fascinating journey of Spinraza's development, a groundbreaking therapy for spinal muscular atrophy.About this EventThe Development of Spinraza, a Disease-Modifying Therapy for Spinal Muscular AtrophyIn collaboration with Ionis Pharmaceuticals and Biogen, we previously developed nusinersen (Spinraza), an antisense oligonucleotide (ASO) that modulates alternative splicing of SMN2 exon 7, restoring normal levels of functional SMN protein in the context of spinal muscular atrophy (SMA). The long duration of action of CNS-administered ASOs like Spinraza allows infrequent dosing by lumbar puncture, providing a feasible and effective approach to treat neurological disorders. I will describe how the convergence of advances in mechanistic studies of alternative splicing regulation, in the molecular genetics of SMA, and in ASO pharmacology, made possible the development of the first disease-modifying therapy for neurodegeneration. The description will cover pre-clinical studies, as well as clinical trials and the real-world impact of Spinraza on SMA patients’ lives.About the speakerProfessor Adrian Krainer, Cold Spring Harbor Laboratory and 2021 Wolf Prize in Medicine LaureateProf Adrian Krainer completed his undergraduate education at Columbia University in 1981, with a B.A. in Biochemistry. He then completed his Ph.D. in Biochemistry at Harvard University in 1986, in the lab of Prof Tom Maniatis. After graduation, he became the first Cold Spring Harbor Fellow, and in 1989 he joined the faculty at Cold Spring Harbor Laboratory. He is currently the St. Giles Foundation Professor at CSHL, and the Deputy Director of Research of the CSHL Cancer Center. He is also a co-founder and Director of Stoke Therapeutics.Adrian's lab at CSHL uses multidisciplinary approaches to elucidate pre-mRNA splicing mechanisms and alternative splicing regulation. An important goal is to apply this knowledge to uncover the roles of defective splicing in genetic diseases and cancer. The lab also uses antisense technology to develop novel therapeutics that target pre-mRNA or mRNA to modulate gene expression post-transcriptionally. One notable application—in collaboration with Ionis Pharmaceuticals and Biogen—was the development of the antisense oligonucleotide nusinersen (Spinraza), which became the first approved drug for spinal muscular atrophy, a severe genetic disease that causes motor-neuron degeneration. Research from the Krainer lab has also implicated splicing alterations in cancer, and his lab is currently pursuing antisense-therapeutic approaches in the context of various cancers.Adrian is a recipient of the Albany Medical Center Prize in Medicine and Biomedical Research, the Wolf Prize in Medicine, the Life Sciences Breakthrough Prize, the RNA Society’s Lifetime Achievement Award, the Reemtsma Foundation International Prize in Translational Neuroscience, the Speiser Award in Pharmaceutical Sciences, the Ross Prize in Molecular Medicine, the Gabbay Award in Biotechnology and Medicine, the Takeda Pharmaceuticals Innovators in Science Senior Scientist Award in Rare Diseases, and the Watanabe Prize in Translational Research. He served as President of the RNA Society, and is a member of the National Academy of Sciences, the National Academy of Medicine, the National Academy of Inventors, and the American Academy of Arts &amp; Sciences.
- **Event URL**: https://allevents.in/jurong/the-development-of-spinraza-a-disease-modifying-therapy-for-sma/100001980438801768
- **Event Categories**: art
- **Interested Audience**: 
  - total_interested_count: 0

## Ticket Details

- **Ticket URL**: https://consumer.pxf.io/c/1339408/3151484/39691?partnerpropertyid=1191892&MediaPartnerPropertyId=1191892&svlink=14105511&level=1&u=https%3A%2F%2Fwww.eventbrite.com%2Fe%2Fthe-development-of-spinraza-a-disease-modifying-therapy-for-sma-tickets-1980438801768
- **Ticket Price Range**: min: 0, max: 0, currency: SGD

## Event venue details

- **city**: Jurong
- **state**: SG
- **country**: Singapore
- **location**: Tan Chin Tuan Lecture Theatre
- **lat**: 1.344087
- **long**: 103.6824955
- **full address**: Tan Chin Tuan Lecture Theatre, 50 Nanyang Avenue, Jurong, Singapore

## Event Organizer details

- **affiliate_id**: 10
- **organizer**: Fellowships & Awards (Research Support Office) (https://allevents.in/org/fellowships-and-awards-research-support-office/27384421)

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## FAQs

- **Q**: When is the event happening?
  - **A:** Tue, 03 Feb, 2026 at 05:00 pm
- **Q**: Where is the event happening?
  - **A:** Tan Chin Tuan Lecture Theatre, 50 Nanyang Avenue, Jurong, Singapore
- **Q**: What is the ticket price?
  - **A:** starting from 0 up to 0 SGD
- **Q**: Who is organizing the event?
  - **A:** Fellowships & Awards (Research Support Office)
- **Q**: What type of event is this?
  - **A:** art
- **Q**: Where can I find ticket details about The Development of Spinraza, a Disease-Modifying Therapy for SMA ?
  - **A:** You can find ticket details about this event on AllEvents.

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