Regulatory and Manufacturing Challenges in Gene Therapy

Issue on Jul-27 : Regulatory and Manufacturing Challenges in Gene Therapy

The dominant theme across the biotech news is the significant regulatory and manufacturing hurdles faced by gene therapy developers, exemplified by Sarepta Therapeutics’ setbacks including FDA rejections and patient safety concerns. These challenges impact clinical progress, investor confidence, and company valuations, highlighting the critical need for robust manufacturing processes and regulatory compliance in advancing cutting-edge gene therapies.

Sarepta Therapeutics faces significant challenges following FDA rejection of its gene therapy for Sanfilippo syndrome due to manufacturing issues, alongside patient deaths linked to its DMD gene therapy Elevidys, leading to CEO suspending full-year guidance and workforce layoffs. ( score: 6 ) link.

Krystal Biotech achieved regulatory approval in Japan for its dystrophic epidermolysis bullosa treatment VYJUVEK®, marking a key milestone in rare disease therapeutics and maintaining strong institutional investor interest. ( score: 5 ) link.

BB Biotech AG outperformed the Nasdaq Biotech Index in Q2 2025 despite currency headwinds, driven by strategic portfolio management focusing on mid-cap biotech firms with promising clinical pipelines and recent approvals. ( score: 5 ) link.

Krystal Biotech maintains strong institutional investor interest and recently received regulatory approval in Japan for its rare disease treatment VYJUVEK®. ( score: 5 ) link.

Gilead Sciences partnered with the Academy of Oncology Nurse & Patient Navigators to launch an oncology navigation grant program, aiming to enhance cancer patient support and care strategies. ( score: 3 ) link.

GSK encountered regulatory setbacks with its blood cancer drug Blenrep facing FDA rejection due to ocular toxicities, and the drug was withdrawn from the market after failing confirmatory trials. ( score: 3 ) link.

Moderna received positive EMA opinion for its updated COVID-19 vaccine targeting new variants but faced FDA criticism over insufficient evidence for its pediatric COVID vaccine, reflecting ongoing regulatory scrutiny. ( score: 3 ) link.

Merck & Co. completed a $10 billion acquisition of Verona Pharma, strengthening its respiratory therapeutics pipeline and preparing for upcoming patent expirations with potential further acquisitions. ( score: 3 ) link.

AstraZeneca is advancing obesity therapeutics beyond GLP-1 receptor agonists, investing $50 billion in U.S. manufacturing and R&D, and preparing to launch a self-administered C5 inhibitor to compete in biologics. ( score: 3 ) link.

Portage Biotech reported significant reduction in net loss for fiscal 2025 and is advancing its clinical-stage immuno-oncology pipeline. ( score: 3 ) link.

Taiho Oncology is highlighted for its pivotal REZILIENT1 Phase 1/2 clinical trial data published in the Journal of Clinical Oncology, marking significant progress in oncology treatment development. ( score: 2 ) link.

Cullinan Therapeutics announced positive clinical trial results from the REZILIENT1 Phase 1/2 study, indicating advancement in cancer therapeutics alongside Taiho Oncology. ( score: 2 ) link.

Bambusa Therapeutics initiated a Phase 1 clinical trial for BBT002, a novel bispecific antibody platform targeting inflammatory and immunological conditions, supported by FDA IND clearance. ( score: 2 ) link.

BioLineRx presented new pilot phase data from a Phase 2 combination trial of Motixafortide in first-line pancreatic cancer at ASCO 2025, underscoring its oncology pipeline progress. ( score: 2 ) link.

Adcentrx Therapeutics received FDA Fast Track designation for its Nectin-4 antibody-drug conjugate targeting advanced cervical cancer, accelerating its clinical development. ( score: 2 ) link.

Novavax is focusing on R&D through a partnership with Sanofi, offloading vaccine costs, and adapting to regulatory frameworks for COVID-19 vaccines amid evolving pandemic response. ( score: 2 ) link.

Trinity Biotech is developing CGM+, an AI-powered continuous glucose monitoring system integrating multi-sensor data for personalized healthcare, targeting a 2026 commercial launch. ( score: 2 ) link.

Ionis Pharmaceuticals is progressing with promising clinical data in hereditary angioedema and other serious conditions, contributing to BB Biotech’s portfolio strength. ( score: 2 ) link.

Vertex Pharmaceuticals reported clinical breakthroughs in type 1 diabetes and other indications, reinforcing its position in innovative biotech pipelines. ( score: 2 ) link.

Apellis Pharmaceuticals is a leader in complement science, developing targeted C3 therapies for rare, retinal, and neurological diseases, with active investor engagement and recent financial reporting. ( score: 2 ) link.

Liminatus Pharma is exploring the formation of a digital asset investment subsidiary to diversify treasury management while continuing its focus on cancer immunotherapies. ( score: 2 ) link.

Adcentrx Therapeutics received FDA Fast Track designation for its antibody-drug conjugate targeting advanced cervical cancer, accelerating clinical development. ( score: 2 ) link.

BioNTech continues to innovate in cancer therapeutics and COVID-19 vaccines but has recently reduced staff in Maryland as part of strategic reorganization. ( score: 2 ) link.

Sino Biopharm acquired Shanghai-based LaNova Medicines, strengthening its oncology pipeline and domestic biotech capabilities. ( score: 2 ) link.

CirCode Biomed achieved FDA clearance for its IND application of HM2002, the world’s first circular RNA drug tested in patients, marking a breakthrough in RNA-based therapeutics. ( score: 1 ) link.

Transcripta Bio is collaborating with Microsoft Research to leverage artificial intelligence for disease-gene discovery, exemplifying the integration of AI in biotech research. ( score: 1 ) link.

Creatv Bio is advancing monitoring of PD-L1 upregulation in metastatic breast cancer, contributing to precision oncology diagnostics. ( score: 1 ) link.

CollPlant secured recent funding to support its development of regenerative medicine products, emphasizing its role in tissue engineering and bioprinting. ( score: 1 ) link.

Astellas Pharma entered a licensing deal for an antibody-drug conjugate targeting CLDN18.2, expanding its oncology portfolio. ( score: 1 ) link.

Sun Pharmaceutical Industries acquired Checkpoint Therapeutics, strengthening its position in oncology therapeutics. ( score: 1 ) link.

BioAtla reported progress in developing antibody therapeutics for solid tumors, highlighting its focus on innovative cancer treatments. ( score: 1 ) link.

Amplifica announced positive clinical outcomes for its treatment of androgenetic alopecia, indicating progress in dermatological therapeutics. ( score: 1 ) link.

Neurogene received regulatory approval for its Rett syndrome trial design, advancing gene therapy development for neuropsychiatric disorders. ( score: 1 ) link.

ImmunityBio reported a 60% revenue increase in Q2 2025, supported by recent equity financing, indicating growth in immunotherapy development. ( score: 1 ) link.

Avalyn Pharma closed a $100 million Series D funding round to advance inhaled therapies for lung diseases, supported by major institutional investors. ( score: 1 ) link.

Castle Biosciences CEO Derek Maetzold was recognized as a Most Admired CEO, reflecting leadership excellence in the biotech sector. ( score: 1 ) link.

Acadia Pharmaceuticals is recognized for strong growth and solid profitability, with a focus on affordable growth strategies in biotech. ( score: 1 ) link.