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BioWorld - Thursday, January 1, 2026
Home » Topics » Drugs » Gene therapy

Gene therapy
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Knee pain illustration

Juniper Biologics acquires gene therapy for knee osteoarthritis in $600M deal from Kolon Life Science

April 19, 2022
By David Ho and Gina Lee
Through a $600 million deal, Juniper Biologics Pte. Ltd. has acquired rights from Kolon Life Science Inc. to develop and commercialize TG-C LD (Tissuegene-C low dose) for the treatment of knee osteoarthritis. Singapore-based Juniper will hold the rights for Asia Pacific, the Middle East and Africa and be responsible for developing and commercializing the candidate within those regions.
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Knee pain illustration

Juniper Biologics acquires gene therapy for knee osteoarthritis in $600M deal from Kolon Life Science

April 14, 2022
By David Ho and Gina Lee
Through a $600 million deal, Juniper Biologics Pte. Ltd. has acquired rights from Kolon Life Science Inc. to develop and commercialize TG-C LD (Tissuegene-C low dose) for the treatment of knee osteoarthritis. Singapore-based Juniper will hold the rights for Asia Pacific, the Middle East and Africa and be responsible for developing and commercializing the candidate within those regions.
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Handshake with DNA, molecules

Close to the bone marrow: Actinium in a $452M deal with Immedica

April 12, 2022
By Lee Landenberger
Actinium Pharmaceuticals Inc. is receiving an up-front $35 million with a possible $417 million in regulatory and commercial milestones in its new license and supply agreement with Immedica Pharma AB, of Stockholm. Immedica is getting the exclusive European, Middle Eastern and North African rights to Iomab-B, an antibody radiation conjugate comprising apamistamab, a CD45-targeting antibody, and the radioisotope iodine-131. Actinium is eligible for royalties in the mid-20% range on net sales.
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Third sacker: Bluebird plots major-league layoffs to stay in gene therapy game

April 5, 2022
By Randy Osborne
Bluebird Bio Inc. became the latest in a spate of gene therapy firms to disclose restructuring plans, as the company aims to save $160 million over the next two years, saying goodbye to about a third of its workforce. It’s the other shoe to drop after Cambridge, Mass.-based Bluebird rattled Wall Street with phraseology in the firm’s fourth-quarter earnings report March 4 that expressed “substantial doubt” regarding whether operations could go on.
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Spotlight raises $36.5M to advance non-viral gene editing tech, lead I-O program

March 22, 2022
By Michael Fitzhugh
Spotlight Therapeutics Inc., a company developing cell-targeted in vivo CRISPR gene editing biologics, has raised $36.5 million in series B financing to support advancement of its first-in-class immuno-oncology program and further applications of its in-house technology platform. The financing round was co-led by new investors GordonMD Global Investments and Epiq Capital Group, with participation from Magnetic Ventures, as well as existing investors GV, Emerson Collective and others.
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Brain and DNA

Novartis options Voyager AAV capsids for CNS gene therapies in potential $1.7B-plus deal

March 8, 2022
By Michael Fitzhugh
Novartis AG, an early and active player in bringing gene therapies to market, has agreed to pay Voyager Therapeutics Inc. $54 million up front and up to $1.7 billion in fees and milestone payments for options to license up to five next-generation adeno-associated virus (AAV) capsids to use as gene therapy vectors for neurological diseases.
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Rafiq Hasan, CEO, Complement Therapeutics
Newco news

Complement Therapeutics emerges from stealth with plan to take on untreatable eye disease

Feb. 23, 2022
By Richard Staines
U.K. biotech Complement Therapeutics Ltd has come out of stealth mode with €5 million ($5.7 million) in seed funding to tackle complement-related diseases, initially targeting the currently untreatable condition geographic atrophy due to dry age-related macular degeneration.
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Splicebio raises record series A to tackle AAV packing problem

Feb. 16, 2022
By Nuala Moran
LONDON – In the largest-ever series A for a Spanish biotech, Splicebio S.L. has raised €50 million (US$56.9 million) to apply its protein splicing technology to the delivery of large genes that do not fit into existing vectors. The company claims its approach will overcome the capacity constraints of adeno-associated viral vectors (AAVs), by splitting genes into parcels and reconstituting the proteins they express in vivo.
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DNA in drug capsules

NICE backs Orchard rare disease drug after further cut to $3.9M list price

Feb. 4, 2022
By Richard Staines
In one of its familiar U-turns, the U.K. National Institute for Health and Care Excellence (NICE) has recommended NHS England should fund a rare disease gene therapy from Orchard Therapeutics plc, considered to be the world’s most expensive drug. The list price for Libmeldy (atidarsagene autotemcel) in England and Wales is £2,875,000 (US$3.9 million), making it the most expensive drug that NICE has ever evaluated.
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Sio Gene drops a program and its CEO

Feb. 1, 2022
By Lee Landenberger
Sio Gene Therapies Inc. is retrenching the business as it terminated the company’s AXO-Lenti-PD license agreement with Oxford Biomedica plc for treating Parkinson’s disease. Also, Sio’s CEO, Pavan Cheruvu, is leaving the company. Sio said it is deprioritizing its Parkinson’s disease program due to several factors, including resource requirements and development timelines “to reach meaningful value inflection for the program and an increasingly challenging market and regulatory environment” for the indication.
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