Insights provided by DeciBio, a strategy consultancy focused on the life science and biopharma industry.
Highlights & Summary
Intro
As we enter the latter half of 2025, regulatory updates, such as FDA orphan drug designations and priority reviews, remain highly relevant across the next-generation therapeutics landscape.
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Cell Therapy
- Mustang Bio Secures Orphan Drug Designation from FDA for CART Therapy in Astrocytoma and Glioblastoma | Regulatory
- Cellipont and CellVax Expand Partnership to Advance cGMP Manufacturing of Personalized Immunotherapy FKGI101 | Partnership
Gene Therapy
- Ultragenyx Receives CRL for Sanfilippo Candidate | Regulatory
- Alexion Initiates Partnership for Novel Capsids | Partnership
- Sarepta’s Rollercoaster July | Regulatory
- Solid Biosciences Receives Fast Track Designation | Regulatory
Oligo Therapy
- Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Duchenne Muscular Dystrophy Drug | Regulatory
- AusperBio Receives CDE Clearance to Advance AHB-137 to Phase III for Chronic Hepatitis B | Clinical Trial
Antibody-Drug Conjugates
- Biokin Posts Phase 3 Win for Bispecific ADC | Clinical Trial
Cell Therapy
Mustang Bio Secures Orphan Drug Designation from FDA for CART Therapy in Astrocytoma and Glioblastoma | Regulatory
Mustang Bio announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to MB‑101, an IL13Rα2-targeted CAR‑T cell therapy, for the treatment of recurrent diffuse and anaplastic astrocytoma and glioblastoma. In a Phase 1 trial published in Nature Medicine, MB‑101 was well-tolerated and achieved stable disease or better in 50% of evaluable patients. The designation builds on a prior Orphan Drug status for MB‑108, an HSV‑1 oncolytic virus, which Mustang plans to combine with MB‑101 as part of its MB‑109 regimen. Clinical trials for MB‑101 at City of Hope and for MB‑108 at UAB are ongoing, though Mustang noted that further development of MB‑109 will depend on additional funding or a strategic partnership.
Cellipont and CellVax Expand Partnership to Advance cGMP Manufacturing of Personalized Immunotherapy FKGI101 | Partnership
Cellipont Bioservices and CellVax Therapeutics have expanded their strategic partnership to support cGMP manufacturing of FKGI101, CellVax’s personalized immunotherapy candidate for gastrointestinal cancers. The collaboration builds on an earlier agreement and enables technology transfer and GMP production of the cell-based therapy at Cellipont’s Carlsbad, CA facility. FKGI101 is an autologous therapy designed to stimulate targeted immune responses against GI tumor antigens and is currently in late preclinical development. Leadership from both companies emphasized the critical role of scalable, quality-focused manufacturing in accelerating the clinical translation of personalized immunotherapies.
Atara’s Allogeneic T-Cell Therapy tab-cel Receives FDA Priority Review for EBV+ PTLD | Regulatory
Atara Biotherapeutics announced that the FDA has accepted its resubmitted BLA for tabelecleucel (tabcel), an allogeneic T-cell immunotherapy for EpsteinBarr virus–positive post-transplant lymphoproliferative disease (EBV+ PTLD), granting the application Priority Review with a PDUFA date of January 10, 2026. If approved, tabcel would become the first FDA-authorized treatment for EBV+ PTLD, a rare and life-threatening condition with no current approved therapies. The resubmission follows a prior Complete Response Letter citing only a third-party manufacturing inspection issue, with no additional clinical data requested. The pivotal ALLELE trial reported a 48.8% objective response rate and a manageable safety profile, supported by clinical experience across over 430 patients.
Adaptimmune Therapeutics has entered into a definitive agreement to sell its commercial and late-stage cell therapy portfolio—including TECELRA (its first FDA-approved TCR T-cell therapy), letecel (with approval anticipated in 2026), afamicel, and uzacel—to US WorldMeds for $55 million in cash upfront, plus up to $30 million in potential milestone payments. The deal includes the transfer of associated intellectual property and U.S.-based employees, while Adaptimmune will retain rights to its preclinical programs, including PRAME, CD70, and allogeneic assets. US WorldMeds has committed to maintaining uninterrupted access to TECELRA and intends to advance letecel toward commercialization. Adaptimmune will undergo a restructuring to focus on its early-stage pipeline.
SCG’s HBV-Specific TCR-T Therapy Receives China NMPA IND Clearance | Regulatory
SCG Cell Therapy announced that China’s National Medical Products Administration has cleared the Investigational New Drug application for SCG101V—marking the first-ever cell therapy authorized to enter a Phase 1/2 clinical trial specifically for chronic hepatitis B infection. SCG101 is engineered to eradicate HBV cccDNA and integrated viral DNA within infected hepatocytes—the persistent reservoirs of infection that current antivirals cannot fully address. In prior Phase 1 data from patients with HBV-related hepatocellular carcinoma, SCG101 demonstrated rapid and durable virologic activity: 94% of patients experienced a decline in HBsAg within 28 days, with levels remaining below 100 IU/mL for up to a year, and 23% achieved complete HBsAg loss—a key functional benchmark. The company emphasized that this IND clearance not only validates their dual antiviral and antitumor strategy but also marks a historic milestone in advancing toward a long-term functional cure for chronic hepatitis B.
Gene Therapy
Ultragenyx Receives CRL for Sanfilippo Candidate | Regulatory
Ultragenyx announced that the FDA has issued a Complete Response Letter (CRL) for its BLA for UX111, an AAV gene therapy for Sanfilippo syndrome type A (MPS IIIA), citing chemistry, manufacturing, and controls (CMC) and facility-related observations. While the clinical data were deemed robust with no noted issues, the company plans to address the manufacturing concerns and resubmit the BLA, potentially delaying approval to 2026.
Alexion Initiates Partnership for Novel Capsids | Partnership
AstraZeneca’s rare disease unit Alexion has signed a licensing deal worth up to $825 million with Japan’s JCR Pharmaceuticals to access its JUST-AAV capsid platform for up to five genomic medicine programs. The deal includes an undisclosed upfront payment, up to $225 million in development and regulatory milestones, $600 million in sales-based milestones, and tiered royalties, expanding an ongoing partnership focused on AAV gene therapy and oligonucleotide development.
Sarepta’s Rollercoaster July | Regulatory
Over the past month, Sarepta Therapeutics' Duchenne muscular dystrophy gene therapy, Elevidys, came under increased FDA scrutiny following reports of three patient deaths—two in Elevidys-treated individuals and one in a related trial for a limb-girdle muscular dystrophy gene therapy. In response, the FDA requested Sarepta suspend Elevidys distribution and placed a partial clinical hold on relevant trials. Sarepta initially resisted, continuing shipments to ambulatory patients while pausing access for non-ambulatory individuals, citing no new safety signals in the former group. However, the company later agreed to halt all shipments. After reviewing the most recent death and determining it was not caused by Elevidys, the FDA lifted its voluntary hold for ambulatory patients, allowing the therapy to return to the U.S. market for that group while maintaining the pause for others.
Rocket Reprioritizes Pipeline and Receives RMAT Designation | Regulatory
Rocket Pharmaceuticals has announced a corporate restructuring and pipeline prioritization to focus resources on its AAV-based cardiovascular gene therapy programs, including those for Danon disease, BAG3-associated dilated cardiomyopathy, and PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), the last of which recently received RMAT designation from the FDA. The company is reducing its workforce by 30% and delaying programs in Fanconi Anemia and Pyruvate Kinase Deficiency to extend its operational runway into Q2 2027. The RMAT designation for RP-A601, Rocket’s gene therapy for PKP2-ACM, follows encouraging Phase 1 data showing increased PKP2 protein expression, improved cardiac function, and a favorable safety profile.
Solid Biosciences Receives Fast Track Designation | Regulatory
Solid Biosciences has received FDA Fast Track designation for SGT-501, its AAV-based gene therapy candidate for catecholaminergic polymorphic ventricular tachycardia (CPVT), a rare inherited heart rhythm disorder with no approved treatments. The therapy delivers a functional CASQ2 gene to cardiac cells to stabilize calcium signaling and correct the underlying cause of arrhythmias. IND clearance was granted earlier in July, and a Phase 1b trial is expected to begin in Q4 2025. SGT-501 has also received Orphan Drug and Rare Pediatric Disease designations.
Oligo
Sarepta Therapeutics Announces Strategic Restructuring and Pipeline Prioritization | Personnel
Sarepta Therapeutics announced a strategic restructuring in July, including a 36% workforce reduction, affecting approximately 500 employees, to address financial pressures and support long-term sustainability. The company will prioritize its siRNA platform, advancing programs in facioscapulohumeral muscular dystrophy, myotonic dystrophy type 1, spinocerebellar ataxia type 2, idiopathic pulmonary fibrosis, and Huntington’s disease, while pausing most limb-girdle muscular dystrophy gene therapy programs except for SRP-9003. Expected annual cost savings are approximately $400 million by 2026, lowering non-GAAP R&D and SG&A expenses to $800–900 million per year. The decision follows increased regulatory scrutiny after the addition of a black box warning to Elevidys.
BioSpring Breaks Ground on Major Offenbach Nucleic Acid API Facility | Manufacturing
BioSpring has begun construction of a 164,000-square-foot GMP manufacturing facility in Offenbach, Germany for DNA- and RNA-based active pharmaceutical ingredients (APIs). The plant will include interconnected manufacturing and warehouse space, quality control offices, a central glass atrium, and an external solvent tank farm, with plans to create about 200 new jobs by the end of 2027. The investment, valued at several hundred million euros, enables BioSpring to manufacture oligonucleotides and other nucleic acids at the ton scale to meet the rising demand for therapeutic and vaccine applications in oncology, neurology, rare genetic, and metabolic diseases.
Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Duchenne Muscular Dystrophy Drug | Regulatory
Avidity Biosciences announced that the FDA has granted Breakthrough Therapy Designation to delpacibart zotadirsen (DEL-ZOTA) for the treatment of Duchenne muscular dystrophy (DMD) in patients whose disease is amenable to exon 44 skipping. The designation was based on data from the Phase 1/2 EXPLORE44 trial, in which DEL-ZOTA demonstrated robust exon 44 skipping, increased dystrophin protein production in muscle tissue, and improvements in key clinical endpoints. DEL-ZOTA is an antibody oligonucleotide conjugate (AOC) engineered to enhance delivery of the therapeutic oligonucleotide to muscle cells by targeting the transferrin receptor. In the trial, the therapy was well tolerated, with no serious adverse events or discontinuations due to treatment-related adverse effects.
Centivax Raises $45M Series A to Advance Universal Flu Vaccine | Financing
Centivax announced the close of an oversubscribed $45 million Series A round led by Future Ventures, with participation from NFX, BOLD Capital Partners, Kendall Capital Partners, Amplify Bio, Base4 Capital, and other investors. The funding enables Centivax to advance its universal flu vaccine candidate into first-in-human clinical studies and to expand its universal immunity portfolio addressing pathogens such as RSV, herpesviruses, HIV, malaria, and more. Centivax’s unique technology for designing immune therapies supports its main flu vaccine candidate, which aims to address the global flu market worth over $7 billion. Dr. Emilio Emini, former CEO of the Gates Medical Research Institute and a major contributor to vaccine development at Pfizer and Merck, has joined Centivax’s Board of Directors as part of this financing. In addition to venture funding, Centivax has secured $24 million in non-dilutive awards from organizations including the Gates Foundation, CEPI, NIH, and the U.S. military.
AusperBio Receives CDE Clearance to Advance AHB-137 to Phase III for Chronic Hepatitis B | Clinical Trial
AusperBio Therapeutics announced that the China Center for Drug Evaluation (CDE) has approved initiation of a Phase III randomized, double-blind, multicenter clinical trial for AHB-137, a novel antisense oligonucleotide, in HBeAg-negative chronic hepatitis B (CHB) patients on stable nucleos(t)ide analogue therapy. The 24-week study will evaluate the efficacy and safety of AHB-137 at a 300mg dose in this population, addressing a disease that affects approximately 290 million people worldwide, including 75 million in China, with high risks of cirrhosis and hepatocellular carcinoma. AHB-137 received Breakthrough Therapy Designation from the China CDE in July 2024 based on data demonstrating significant improvements in functional cure rates compared to current treatment options.
ADCs
Simtra BioPharma Solutions Announces Purchase to Expand Manufacturing Capacity | Manufacturing
Simtra BioPharma Solutions has purchased a 65-acre property near its Bloomington, Indiana facility from Cook Group, providing over 300,000 square feet of expansion space for injectable medicine manufacturing. This acquisition supports Simtra’s goal of becoming the first CDMO in the U.S. to offer commercial-scale manufacturing of antibody-drug conjugate (ADC) drug products. The company is considering the installation of isolator-based vial and prefilled syringe filling lines using a build-to-suit model based on customer and product requirements. The site will complement Simtra’s $250 million expansion in Bloomington and recent facility investments in Germany.
Biokin Posts Phase 3 Win for Bispecific ADC | Clinical Trial
Biokin has announced that a Phase 3 trial of its bispecific ADC candidate izalontamab brengitecan met at least one primary endpoint in patients with recurrent or metastatic nasopharyngeal carcinoma after prior PD1/L1 therapy. Bristol Myers Squibb originated an exChina licensing deal worth up to $8.4 billion, including an $800 million upfront payment in 2023 to secure global rights to the drug. The ADC targets EGFR and HER3 receptors and delivers a cytotoxic payload to tumor cells expressing both markers. BMS is now planning a Phase 2/3 trial in triplenegative breast cancer, while Biokin’s Chinese partner Baili Pharmaceutical is also evaluating the asset in multiple other tumor types.
FDA Flags Concerns Over Eye Safety Risks for GSK’s Blenrep ADC | Regulatory
FDA staff raised concerns about GSK’s blood cancer drug Blenrep, citing risks of eye damage such as blurred vision, photophobia, and dry eyes when used in combination therapies for relapsed or refractory multiple myeloma. Reviewers questioned the benefit-risk profile and noted that the eye safety monitoring and dose adjustments used in trials may be hard to implement in real-world settings. During a July advisory meeting, an FDA panel voted against approving Blenrep in both proposed combination regimens due to safety concerns, limited U.S. patient representation in trials, and trial design issues.
Sutro BioPharma Announces Research Collaboration with the FDA | Partnership
Sutro Biopharma has entered into a research collaboration with the FDA to develop reference materials aimed at advancing regulatory standards and analytical methods for antibody-drug conjugates (ADCs). The initiative will utilize Sutro’s XpressCF® cell-free protein synthesis platform to create well-defined ADCs that can serve as benchmarks for regulatory evaluation. Working with the FDA’s Office of Pharmaceutical Quality, the project will focus on selecting representative antigens, payload-linkers, and conjugation sites based on existing and in-development ADCs. The results of the collaboration will be published publicly and are intended to support more consistent and rigorous regulatory assessments across the ADC field.
DualityBio’s Next Generation HER3 ADC Granted FDA Fast Track Designation | Regulatory
Duality Biologics has received Fast Track Designation from the U.S. FDA for its next-generation HER3-targeting antibody-drug conjugate (ADC), DB-1310. This designation is for treating advanced, unresectable, or metastatic nonsquamous non-small cell lung cancer (nsqNSCLC) with specific EGFR mutations that have progressed after treatment with third-generation EGFR tyrosine kinase inhibitors and platinum-based chemotherapy. Developed using Duality’s proprietary DITAC platform, DB-1310 has shown promising efficacy and a manageable safety profile in early-phase clinical trials. The company aims to accelerate its development to offer a new treatment option for this patient population.