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511,731 Results for "sio gene therapies".
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Gene therapy
Gene Therapies for Hearing Loss Strike an Encouraging Note in Embattled Modality
Eli Lilly and Regeneron are leading the push to treat congenital deafness with gene therapies, seeking a piece of a potential billion-dollar market and banking on local delivery and the small amount of drug required to overcome key safety concerns.
March 23, 2026
·
7 min read
·
Tristan Manalac
Manufacturing
Cell and Gene Therapy Manufacturing Market To Skyrocket to $146B by 2032
Analysts expect the market for manufacturing cell and gene therapies, worth less than $20 billion in 2024, to expand rapidly as approvals drive higher volumes of production.
March 10, 2026
·
3 min read
·
Nick Paul Taylor
Gene editing
Pfizer Advances Beam’s Gene Editor After Pulling Hemophilia Gene Therapy
Pfizer will be responsible for all global clinical and regulatory activities for Beam Therapeutics’ liver-directed gene editor.
February 26, 2026
·
1 min read
·
Tristan Manalac
Press Releases
RenovoRx Announces Clinical Data Supporting the TAMP™ Therapy Platform will be Presented at the SIO 2026 Annual Scientific Meeting
February 5, 2026
·
10 min read
Rare disease
Why Industry Is Questioning FDA’s Data-Sharing Push in Cell and Gene Therapy
After the FDA’s first-ever public listening meeting on data-sharing in the cell and gene therapy space, new draft guidance aims to standardize the practice. But recent decisions call into question whether shared evidence and prior knowledge will accelerate development in rare diseases.
March 12, 2026
·
5 min read
·
Ana Mulero
Sickle cell disease
Sickle Cell Gene Therapies Casgevy and Lyfgenia Still Lacking Traction 2 Years In
The first gene therapies approved to treat sickle cell disease in December 2023 are struggling on the market. But there are glimpses of forward momentum as Vertex and Genetix Bio provide updates.
February 23, 2026
·
6 min read
·
Heather McKenzie
Gene therapy
In Vivo Is Having a Moment as Cell and Gene Therapy Sector Gathers in San Diego
A recurring theme Tuesday morning at Phacilitate’s Advanced Therapies Week was the quickly emerging potential of in vivo approaches to cell and gene therapy—a trend also reflected in recent investments by Eli Lilly and Regeneron.
February 11, 2026
·
4 min read
·
Heather McKenzie
Complete response letters
REGENXBIO’s Hunter Syndrome Gene Therapy Fails To Win FDA Nod
The FDA recommended that REGENXBIO run a new study, treat more patients and include a placebo arm to support a resubmission for the gene therapy RGX-121.
February 10, 2026
·
2 min read
·
Tristan Manalac
Rare disease
UniQure Eyes Phase 3 for Huntington’s Gene Therapy Despite Makary’s Apparent Criticism
Days after FDA Commissioner Marty Makary appeared to malign uniQure’s AMT-130 in an interview with
CNBC
, the agency confirmed to the biotech that a sham surgery–controlled study is needed before submitting the gene therapy for approval.
March 2, 2026
·
3 min read
·
Heather McKenzie
Press Releases
Neural Therapeutics Announces Amendment to SIO Agreement, Concurrent Financing Terms, Shareholder Meeting Date and RSU Grant
March 27, 2026
·
6 min read
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