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FDA Issues Guidance to Accelerate Cell and Gene Therapy Development

FDA Issues Guidance to Accelerate Cell and Gene Therapy Development

The FDA has released draft guidance aimed at expediting the development of cell and gene therapies (CGT) by allowing developers to utilize existing scientific knowledge to streamline regulatory submissions. This initiative is part of a broader effort by the FDA to reduce barriers for CGT sponsors, particularly for diseases with limited treatment options. The guidance encourages the use of publicly available data and established knowledge, such as chemistry, manufacturing, and controls (CMC) data, to facilitate faster approval processes. The FDA's acting director of the Center for Biologics Evaluation and Research, Karim Mikhail, emphasized the importance of this guidance in delivering safe and effective therapies to patients with rare and life-threatening diseases.
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Advanced NanoTherapies Raises $31M for Dual-Drug Vascular Treatment Platform

Advanced NanoTherapies Raises $31M for Dual-Drug Vascular Treatment Platform

Advanced NanoTherapies Raises $31M for Dual-Drug Vascular Treatment Platform
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MEDIPOST Secures FDA Agreement for Phase 3 Study of CARTISTEM in U.S., Advancing Osteoarthritis Treatment

MEDIPOST Secures FDA Agreement for Phase 3 Study of CARTISTEM in U.S., Advancing Osteoarthritis Treatment

MEDIPOST Secures FDA Agreement for Phase 3 Study of CARTISTEM in U.S., Advancing Osteoarthritis Treatment
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Otsuka's VOYXACT Shows Promise in Preserving Kidney Function in IgA Nephropathy

Otsuka's VOYXACT Shows Promise in Preserving Kidney Function in IgA Nephropathy

Otsuka's VOYXACT Shows Promise in Preserving Kidney Function in IgA Nephropathy
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Stakeholders Call for Pause on FDA's National Priority Voucher Program Amid Transparency Concerns

Stakeholders Call for Pause on FDA's National Priority Voucher Program Amid Transparency Concerns

Several representatives from patient, industry, and trade groups have called for a temporary halt to the FDA's Commissioner’s National Priority Voucher (CNPV) program. The program, introduced by former FDA Commissioner Marty Makary in June 2025, aims to expedite drug approvals for companies aligning with national priorities. However, stakeholders have raised concerns about the program's transparency and potential political influence. Janet Krommes, chair of the FDA task force for Doctors for America, emphasized the need for an independent and credible FDA, criticizing the lack of public involvement in the program's creation. The program's criteria for awarding vouchers remain unclear, and there have been allegations of political appointees influencing the review process. In February, Rep. Jake Auchincloss highlighted the secrecy surrounding the program, which has not responded to Congressional information requests.
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MEDIPOST Gains FDA Approval for Phase 3 Study of CARTISTEM in U.S.

MEDIPOST Gains FDA Approval for Phase 3 Study of CARTISTEM in U.S.

MEDIPOST, a biotechnology company specializing in cell therapy, has secured FDA approval to conduct a single pivotal Phase 3 study for its knee osteoarthritis treatment, CARTISTEM, in the U.S. This agreement marks a significant step in MEDIPOST's efforts to bring its treatment to the U.S. and Canadian markets. The company plans to leverage successful Phase 3 data from South Korea and Japan, along with real-world evidence from 550 patients, to support its U.S. clinical development. This strategy aims to expedite the treatment's commercial entry into the U.S. market.
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Acting US FDA chief meets with rare disease groups to mend fences

Acting US FDA chief meets with rare disease groups to mend fences

By Chris Prentice NEW YORK, June 3 (Reuters) - Acting U.S. FDA Commissioner Kyle Diamantas is meeting with rare disease groups on Wednesday, according to groups attending and a government official, as the new chief seeks to repair relations with a sector disappointed by his predecessor.
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Fulcrum Therapeutics Halts Development of Sickle Cell Drug Pociredir Amid Safety Concerns

Fulcrum Therapeutics Halts Development of Sickle Cell Drug Pociredir Amid Safety Concerns

Fulcrum Therapeutics Halts Development of Sickle Cell Drug Pociredir Amid Safety Concerns
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Advanced NanoTherapies Secures $31M Series B Funding for Innovative Vascular Treatment

Advanced NanoTherapies Secures $31M Series B Funding for Innovative Vascular Treatment

Advanced NanoTherapies Secures $31M Series B Funding for Innovative Vascular Treatment
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Moonlight Therapeutics Launches Phase I Trial for Peanut Allergy Treatment MOON101

Moonlight Therapeutics Launches Phase I Trial for Peanut Allergy Treatment MOON101

Moonlight Therapeutics Launches Phase I Trial for Peanut Allergy Treatment MOON101
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FDA Approves CAVHANZA Tablets for Enhanced Ph+ CML Treatment Flexibility

FDA Approves CAVHANZA Tablets for Enhanced Ph+ CML Treatment Flexibility

FDA Approves CAVHANZA Tablets for Enhanced Ph+ CML Treatment Flexibility
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US FDA to review AI-based tool to predict drug-related liver damage

US FDA to review AI-based tool to predict drug-related liver damage

June 3 (Reuters) - The U.S. FDA's Center for Drug Evaluation and Research said on Wednesday it has accepted a letter of intent for an artificial intelligence-based drug development tool designed to help predict drug-induced liver injury.