FDA Issues Draft Guidance for Gene Therapies
Written by Emily J. Thompson, Senior Investment Analyst
Updated: Jun 02 2026
0mins
The U.S. Food and Drug Administration said it has issued draft guidance to help developers bring promising gene therapies to patients more efficiently by making greater use of existing scientific and regulatory knowledge. When finalized, the guidance will outline how sponsors can use publicly available information and established platform knowledge, including chemistry, manufacturing and controls data, nonclinical study results and clinical information, to streamline regulatory submissions for human gene therapy products that use genome editing in human somatic cells. "Today's action reflects the FDA's commitment to get safe and effective cell and gene therapies to patients faster, particularly those living with rare and life-threatening diseases who have few or no other treatment options," said Karim Mikhail, B. Pharm., M.S., Acting Director of the Center for Biologics Evaluation and Research. "By providing information on how companies may build on what is already known we are accelerating innovation without compromising the rigorous scientific standards that patients and the public depend on.Ultimately, this is about making sure that the promise of gene therapy reaches the patients who need it most, as quickly and safely as possible." This draft guidance supports the development of a wide range of cell and gene therapy products, including those that use genome editing, and is part of a broader set of complementary FDA actions in this area. Publicly traded large-cap drugmakers include AstraZeneca (AZN), Bristol Myers (BMY), Eli Lilly (LLY), GSK (GSK), Johnson & Johnson (JNJ), Merck (MRK), Novartis (NVS), Pfizer (PFE), Roche (RHHBY) and Sanofi (SNY). Biotech stocks include Replimune (REPL), Moderna (MRNA), Sarepta (SRPT), Xencor (XNCR), PTC Therapeutics (PTCT), Biohaven (BHVN), Edgewise Therapeutics (EWTX), Design Therapeutics (DSGN), Ultragenyx (RARE), Dyne Therapeutics (DYN), Wave Life Sciences (WVE), MeiraGTx (MGTX), uniQure (QURE), Regenxbio (RGNX), Novavax (NVAX) and Lexeo Therapeutics (LXEO).
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Analyst Views on AZN
Wall Street analysts forecast AZN stock price to rise
14 Analyst Rating
13 Buy
0 Hold
1 Sell
Strong Buy
Current: 190.160
Low
157.61
Averages
213.64
High
252.18
Current: 190.160
Low
157.61
Averages
213.64
High
252.18
About AZN
AstraZeneca PLC is a United Kingdom-based science-led biopharmaceutical company. The Company focuses on the discovery, development, and commercialization of prescription medicines. The Company operates across therapy areas, including Oncology; Cardiovascular, Renal and Metabolism (CVRM); Respiratory and Immunology (R&I); Vaccines and Immune Therapies (V&I), and Rare Disease. In the Oncology area, its key products include Tagrisso, Imfinzi, Calquence, Lynparza, and Enhertu. The key products of CVRM area include Farxiga/Forxiga, Brilinta/Brilique, Crestor, and Lokelma. In the R&I area, the key products are Symbicort, Fasenra, Breztri/Trixeo, and Tezspire. In the V&I Therapies area, the products are Beyfortus and FluMist. The products in the Rare Disease area are Ultomiris, Soliris, Strensiq, and Koselugo. It has about 191 projects in its development pipeline, including 19 new molecular entities (NMEs) in the late-stage pipeline. The Company distributes its products in over 125 countries.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
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