Scientist awarded for breakthrough RNA research that led to Spinraza
Adrian R. Krainer wins 2025 Heinrich Wieland Prize
A New York-based scientist has been awarded for his pioneering work that led to the development of Spinraza (nusinersen), the first RNA-based therapy to target the underlying cause of spinal muscular atrophy (SMA).
Adrian R. Krainer, PhD, a professor at Cold Spring Harbor Laboratory, in New York, will formally receive the 2025 Heinrich Wieland Prize from the Boehringer Ingelheim Foundation on Dec. 11 during the foundation’s annual symposium and award ceremony in Munich. The prize includes €250,000 (about $293,000 US) in award money.
Awarded annually, the prize honors leading international scientists whose work has significantly advanced the fields of chemistry, biochemistry, or physiology, with particular relevance to human health.
Krainer known for research on messenger RNA splicing
Krainer’s research on the mechanisms and regulation of messenger RNA (mRNA) splicing — a process by which cells edit genetic messages before using them as templates to produce proteins — provided the scientific rationale behind the first approved disease-modifying therapy for people with SMA.
“It’s an honor to be selected as the 2025 Heinrich Wieland Prize laureate,” Krainer said in a news story from Cold Spring Harbor Laboratory. “I am grateful to the Boehringer Ingelheim Foundation for recognizing the value of my lab’s research on mRNA splicing and RNA-targeted therapeutics. It is very humbling to join such a distinguished group of prior recipients.”
SMA is primarily caused by mutations in the SMN1 gene, which normally provides instructions for producing the survival motor neuron protein, or SMN. With little or no SMN function, the motor neurons that play a key role in controlling movement are lost, leading to progressive muscle weakness.
Another gene, called SMN2, acts as a backup to the SMN1 gene; however, due to a small difference in its DNA sequence, it typically produces only 10% to 15% of fully functional SMN protein, which cannot fully compensate for the loss of SMN1.
Krainer discovered that this was due to a defect occurring during the splicing process, which causes the exclusion of a portion of genetic information called exon 7 from most of the mRNA molecules originating from SMN2. This results in a shortened, less stable, and poorly functional protein.
Spinraza, marketed by Biogen, is designed to boost the production of functional SMN protein made from SMN2 by correcting the faulty splicing process. The therapy is an antisense oligonucleotide, a type of RNA-like molecule that binds to SMN2’s mRNA molecule.
“Adrian Krainer is an exceptional scientist whose groundbreaking insights into fundamental biology have come full circle in their impact on human health,” said Franz-Ulrich Hartl, MD, PhD, chair of the scientific board of trustees that selects the Heinrich Wieland Prize recipients. “He uncovered the molecular mechanism behind a splicing defect in SMA patients and pioneered an entirely new approach to correcting it.”
Spinraza was approved in the U.S. for children and adults in 2016, and in the European Union one year later. A higher-dosing regimen of nusinersen is currently being considered for regulatory approval in the U.S., Europe, Japan, and other locations.
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