A splicing defect in the survival of motor neuron gene (SMN) leads to a deficiency of protein function that results in spinal muscular atrophy. It is the second most common autosomal recessive disease, occurring with a prevalence of 1 to as little as 6,000 births.

Researchers at the University of California at San Diego have used a gene therapy approach to treat pain by specifically suppressing the Nav 1.7 ion channel in the spinal cord, both preventing and reversing pain in several animal models with distinct underlying reasons for pain.

LONDON – The National Health Service (NHS) in the U.K. has agreed to reimburse Zolgensma, the one-off gene therapy treatment for spinal muscular atrophy billed as the most expensive drug in the world, after a confidential deal was struck with Novartis Gene Therapies.

Gene therapy developer Beam Therapeutics Inc. has acquired drug delivery specialist Guide Therapeutics Inc. in a deal valued at up to $440 million, adding yet another piece of the integrated platform CEO John Evans told BioWorld the company is striving to build. Guide's lipid nanoparticle (LNP) screening technology could help medicines developed by Beam and its partners reach new targets beyond the liver, an increasingly important endeavor for gene therapy developers like Beam, seeking to maximize the breadth of their growing portfolios. Terms of the deal included $120 million up front, plus up to $320 million in stock-based technology and product success milestone payments. Shares in Beam (NASDAQ:BEAM) fell 12.6% to $96.48 on Feb. 23.

Ensoma Inc., a gene therapy startup working to drastically simplify the production and delivery of genomic medicines, announced itself Feb. 11 with a $70 million series A financing plus a strategic collaboration with Takeda Pharmaceutical Co. Ltd., which licensed vectors from the company for up to five rare disease targets in a deal that could deliver as much as $1.25 billion in potential payouts. Ensoma's co-founder and seed investor, 5AM Ventures, led the financing. Takeda took a $10 million equity stake as part of the round.

A packaged nanoparticle that delivered a relaxin-encoding gene therapy with microRNA reduced liver fibrosis, researchers in the laboratory of Leaf Huang at the University of North Carolina, Chapel Hill, Eshelman School of Pharmacy described in the Jan. 21, 2021, issue of Nature Nanotechnology.

Sarepta Therapeutics Inc.’s miss on a key phase II ambulatory endpoint in its Duchenne muscular dystrophy (DMD) trial may have been caused by a dramatic disparity in functional ability at baseline among older vs. younger patients afflicted with the progressively worsening disorder. In any case, Wall Street had knives out, carving 51% of the value away from shares (NASDAQ:SRPT) of the Cambridge, Mass.-based firm, which closed at $82.29, a loss of $86.66, or 51%.

A third-quarter progress report from the international advocacy group Alliance for Regenerative Medicine (ARM) has determined that the regenerative medicine and advanced therapy (RMAT) sector established a highwater mark of $15.9 billion in global financings, breaking the previous annual record of $13.5 billion that was set in 2018.