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Frontera Therapeutics Receives FDA Approval for Phase II Study of FT-003 in nAMD
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Frontera Therapeutics, Inc., a Sino-US biopharmaceutical company, has announced that it has received approval from the US Food and Drug Administration (FDA) to conduct a Phase II clinical study for its investigational drug FT-003. This recombinant adeno-associated virus (rAAV) gene therapy is designed for the treatment of neovascular age-related macular…
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Beijing Aims to Build a Leading Cell and Gene Therapy Hub by 2026 with New Action Plan
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The Beijing Municipal Science & Technology Commission, in conjunction with the Administrative Commission of Zhongguancun Science Park, has unveiled the “Beijing Action Plan for Accelerating Collaborative Innovation in Medicine and Health (2024-2026)”. The plan is designed to cement Beijing’s position as a global hub for cell and gene therapy (CGT)…
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Reforgene Medicine’s Gene Editing Therapy RM-101 Clears IND Hurdle for Usher Syndrome Treatment
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Reforgene Medicine, a pioneer in gene editing therapy based in Guangzhou, has secured Investigational New Drug (IND) approval from China’s National Medical Products Administration (NMPA) for its flagship candidate, RM-101. This AAV-based gene editing drug is aimed at treating Usher syndrome, a genetic condition that leads to significant vision impairment…
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Jiangsu Hengrui’s RGL-193 Receives NMPA Greenlight for Parkinson’s Disease Clinical Study
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Jiangsu Hengrui Medicine Co., Ltd, a leading pharmaceutical company based in China and listed as (SHA: 600276), has received approval from the National Medical Products Administration (NMPA) for its Category 1 drug, RGL-193. This marks a significant step forward as the drug advances into clinical studies focused on Parkinson’s disease…
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Belief BioMed Partners with Bayer’s AskBio to Explore New Gene Therapies
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Belief BioMed Inc. (BBM), a Chinese biopharmaceutical company specializing in gene therapy, has announced a strategic partnership with Asklepios BioPharmaceutical, Inc. (AskBio), a wholly-owned subsidiary of Bayer AG. This collaboration aims to harness the potential of new gene therapies. The agreement will see the companies pool their expertise and resources…
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Frontera Therapeutics Receives FDA Approval for Phase II Study of First Chinese rAAV Gene Therapy
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Frontera Therapeutics, Inc., a Sino-US biopharmaceutical company, has announced that it has received approval from the US Food and Drug Administration (FDA) to proceed with a Phase II clinical study for its investigational gene therapy, FT-002. This recombinant adeno-associated virus (rAAV) gene therapy candidate marks a significant milestone as the…
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Itcure Expands Gene Editing Reach with Collaborations in China’s Biotech Sector
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Itcure, a prominent gene editing specialist headquartered in Suzhou, has announced separate strategic collaborations with two domestic biotechnology companies: Lakeshore Biotechnology, a cell and gene therapy (CGT) focused biotech, and Porton Pharma Solutions, Ltd., a CGT contract research organization (CRO) and contract development and manufacturing organization (CDMO). In July, Itcure…
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Eli Lilly Partners with Haya Therapeutics to Tap the Dark Genome for Obesity Treatments
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Eli Lilly & Co. (NYSE: LLY), a leading figure in the pharmaceutical industry, has embarked on a drug discovery partnership with Switzerland’s Haya Therapeutics, a move that underscores its commitment to advancing its anti-obesity portfolio. The collaboration will tap into Haya’s cutting-edge expertise in the dark genome, the largely non-protein-coding…
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Skyline Therapeutics’ Gene Therapy Receives FDA Orphan Drug Designation for Retinitis Pigmentosa Treatment
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Skyline Therapeutics, a biopharmaceutical company based in China, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for its proprietary adeno-associated virus (AAV) gene therapy, SKG1108, for the treatment of retinitis pigmentosa (RP). This innovative therapy utilizes a novel AAV vector, including a groundbreaking capsid…
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AccurEdit Therapeutics’ Gene-Editing Drug ART001 Clears FDA Hurdle for US Clinical Trials
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SUZHOU—AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading Chinese specialist in gene editing therapies, has announced that it has received approval from the US Food and Drug Administration (FDA) for its investigational drug ART001, which targets transthyretin amyloidosis. This milestone marks ART001 as the first in vivo gene-edited drug based on…
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BioMarin to Cut 225 Jobs Amid Challenges in Gene Therapy Commercialization
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US-based BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) is navigating a challenging period marked by difficulties in commercializing its gene therapies. In a recent SEC filing, the company announced plans to reduce its workforce by 225 employees as part of its “organizational redesign efforts.” The restructuring is tied to BioMarin’s revised commercial…
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CNGB Virogin’s VG161 On Track for Breakthrough Designation in China for Advanced Liver Cancer
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BEIJING—CNGB Virogin, a joint venture between Virogin Biotech and China National Biotec Group, is poised to secure a breakthrough therapy designation (BTD) from China’s National Medical Products Administration (NMPA) for its innovative drug VG161. The targeted indication for this recombinant human IL12/15-PDL1B herpes simplex type I oncolytic virus injection is…
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Shanghai BDgene Partners with SPH RD to Develop Hemophilia Treatments Using BDlendi Insulator Technology
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Shanghai BDgene Technology Co., Ltd, a leading gene therapy specialist based in China, has entered into a strategic partnership with SPH RD, a subsidiary of Shanghai Pharmaceuticals Holding Co., Ltd focused on rare diseases and pediatrics. The collaboration centers around BDgene’s innovative BDlendi insulator technology, which is slated for the…
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Shenzhen Salubris Pharmaceuticals Inks Licensing Deal for YolTech Therapeutics’ YOLT-101 Gene Editing Therapy
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Shenzhen Salubris Pharmaceuticals Co., Ltd, a Chinese pharmaceutical company listed on the Shenzhen Stock Exchange (SHE: 002294), has entered into a licensing agreement with fellow Chinese firm YolTech Therapeutics. The agreement grants Salubris exclusive rights to research and development, regulatory filings, manufacturing, and commercial sales of YOLT-101 in mainland China.…
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Jiangsu Simcere Pharmaceutical Gets NMPA Green Light for SIM0508 Clinical Trial in Advanced Solid Tumors
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Jiangsu Simcere Pharmaceutical Co., Ltd, a Chinese pharmaceutical company listed in Hong Kong (HKG: 2096), has announced that it has obtained clinical trial approval from China’s National Medical Products Administration (NMPA) for its pipeline candidate SIM0508. This molecule is a DNA polymerase theta (Pol theta) small molecule inhibitor, which is…
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Gene Cradle Initiates First Regulatory Gene Therapy Study in China for Late-Onset Pompe Disease
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Gene Cradle, a Beijing-based gene therapy specialist, has achieved a milestone by administering its GC301 to the first patient with late-onset Pompe disease (LOPD) in a regulatory study in China. This marks the nation’s first patient dosing of an adeno-associated virus (AAV) gene therapy for LOPD. GC301 is an innovative…
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Chugai Seeks Approval for Elevidys Gene Therapy in Japan, Targeting Duchenne Muscular Dystrophy
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Chugai Pharmaceutical Co., Ltd. (TYO: 4519), a subsidiary of Roche, has submitted a product approval application in Japan for delandistrogene moxeparvovec, marketed globally as Elevidys, a gene therapy aimed at treating Duchenne muscular dystrophy (DMD). The therapy is specifically indicated for ambulatory boys aged 3 to 7 years with DMD…
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HuidaGene Secures Patent for Innovative CRISPR-Cas12i System in China
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HuidaGene (Shanghai) Biotechnology Co., Ltd, a China-based biotech firm, has secured patent approval from the China National Intellectual Property Administration for its proprietary DNA editing system, CRISPR-Cas12i, also known as the Cas12Max system. The patent encompasses the hfCas12Max nuclease, which HuidaGene independently developed, as well as the broader CRISPR-Cas12i system…
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Sangamo Therapeutics and Genentech Join Forces to Develop Genomic Therapies for Neurodegenerative Diseases
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Sangamo Therapeutics, Inc. (NASDAQ: SGMO), a genomic medicine company based in the U.S., has entered into a licensing agreement with Genentech, a subsidiary of Swiss pharmaceutical giant Roche. The deal is aimed at developing intravenous genomic medicines for the treatment of specific neurodegenerative diseases. Sangamo has provided Genentech with an…
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Porton Pharma Solutions Partners with Guangzhou Geneseed Biotech to Advance circRNA Therapies
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Porton Pharma Solutions Ltd, a China-based Contract Development and Manufacturing Organization (CDMO), has entered into a strategic partnership with Guangzhou Geneseed Biotech Co., Ltd., a company specializing in circular RNA (circRNA)-focused CRDMO services. This collaboration aims to expedite the development of innovative circRNA therapies, enhancing technical innovation, clinical translation, and…
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Belief BioMed’s Hemophilia B Gene Therapy BBM-H901 Slated for Priority Review in China
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The Center for Drug Evaluation (CDE) has indicated on its website that the market application for BBM-H901, a gene therapy treatment for hemophilia B developed by Belief BioMed Group (BBM), a Shanghai-based specialist in gene therapy, is on track for priority review. BBM-H901, co-developed with Japan’s Takeda, is an adeno-associated…
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Belief BioMed and Takeda’s Hemophilia B Gene Therapy BBM-H901 Accepted for NMPA Review
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Belief BioMed Group (BBM), a Shanghai-based gene therapy specialist, and Takeda have announced that the National Medical Products Administration (NMPA) has accepted for review the New Drug Application (NDA) for BBM-H901, a gene therapy for hemophilia B. This development marks a significant milestone for the treatment, which is being designed…
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Pfizer’s Hemophilia A Gene Therapy Shows Promise in Phase III AFFINE Study
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Pfizer Inc. (NYSE: PFE), a US pharmaceutical giant, has announced positive topline results from the Phase III AFFINE study for its gene therapy candidate, giroctocogene fitelparvovec, for the treatment of hemophilia A. With these results, Pfizer is now poised to prepare for regulatory submission for this one-time gene therapy, which…
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China’s NMPA Considers Breakthrough Designations for Xiangxue and Innostellar’s Therapies
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Xiangxue Pharmaceutical Co., Ltd (SHE: 300147) and Innostellar Biotherapeutics Co., Ltd have both been noted by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) as potential candidates for breakthrough therapy designations (BTDs). Xiangxue’s TAEST16001, a T-cell receptor TCR affinity-enhanced specific T-cell (TAEST) therapy, and Innostellar’s…
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Shanghai Medicilon and Jiangsu Hengrui Join Forces to Advance Pre-Clinical Drug Evaluation
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Shanghai Medicilon Biomedical Co., Ltd, a leading biopharmaceutical company based in China, has entered into a strategic partnership with Jiangsu Hengrui Medicine Co., Ltd (SHA: 600276), a prominent player in the global pharmaceutical industry. This collaboration aims to leverage Medicilon’s pre-clinical research and development expertise with Hengrui’s innovative drive and…
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AccurEdit Therapeutics Receives CDE Approval for Gene Editing Drug ART001 Targeting Amyloidosis
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AccurEdit Therapeutics (Suzhou) Co., Ltd, a leading specialist in gene editing therapies based in China, has received approval from the Center for Drug Evaluation (CDE) for its investigational drug ART001. This drug is designed to target transthyroxine protein amyloidosis, a severe genetic disorder. ART001 marks a significant advancement in China’s…
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China Issues Ethical Guidelines for Human Genome Editing, Prohibits Germline Editing
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China’s Ministry of Science and Technology (MOST) has issued the Ethical Guidelines for Human Genome Editing Research, establishing a framework to regulate such activities and ensure their responsible development. The guidelines place a stringent prohibition on germline editing, which involves genetic modifications that could be passed down to future generations.…
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Jiangsu Hengrui’s Subsidiary Conducts First Surgical Dosing in Parkinson’s Gene Therapy Clinical Trial
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Jiangsu Hengrui Medicine Co., Ltd (SHA: 600276), a leading pharmaceutical company in China, has announced that its subsidiary, Shanghai Regenelead Therapies Co., Ltd., has conducted the first surgical dosing in a clinical study for its dual adeno-associated virus (AAV) gene therapy, RGL-193, aimed at treating Parkinson’s disease (PD). The procedure…
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Shanghai Vitalgen BioPharma Launches Phase I/II Gene Therapy Trial for Parkinson’s Disease
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Shanghai Vitalgen BioPharma Co., Ltd, a biopharmaceutical company based in China, has initiated a Phase I/II clinical study for its gene therapy candidate VGN-R09b at Huashan Hospital, targeting primary Parkinson’s disease (PD). VGN-R09b received clearance to proceed with clinical trials in China for primary PD and aromatic L-amino acid decarboxylase…
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Novo Nordisk Moves to Acquire 2seventy bio’s Hemophilia A Program and Gene-Editing Technology
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Novo Nordisk (NYSE: NVO; CPH: NOVO-B), a leading Danish biopharmaceutical company, is transitioning from a partnership to an acquisition deal with U.S. biotech firm 2seventy bio Inc. (NASDAQ: TSVT) for the development of a hemophilia A drug candidate. The companies have entered into an asset purchase agreement that will see…
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StarryGene Initiates Phase I Clinical Trial for XMVA09 Gene Therapy in wAMD
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StarryGene, a biotech company specializing in gene therapy and based in Hefei, China, has announced that the first patient has been dosed in a Phase I clinical study for its gene therapy XMVA09 injection, which is designed to treat wet age-related macular degeneration (wAMD). XMVA09 is positioned as the world’s…
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Roche Secures RNA Exon Editing Tech for Neurological Diseases with Ascidian Deal
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Roche (SWX: ROG; OTCMKTS: RHHBY), a Swiss pharmaceutical giant, is expanding its footprint in the RNA drug development sector through a strategic partnership and licensing agreement with Ascidian Therapeutics, a U.S.-based biotechnology company. The collaboration aims to discover and develop RNA exon editing therapeutics for the treatment of neurological disorders.…
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Pfizer’s Gene Therapy for Duchenne Muscular Dystrophy Misses Late-Stage Trial Endpoints
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Pfizer Inc. (NYSE: PFE) has announced that a late-stage clinical trial for its investigational gene therapy, fordadistrogene movaparvovec, in children aged 4 to 7 with Duchenne muscular dystrophy (DMD) failed to meet its primary endpoint of improving motor function. Additionally, secondary endpoints related to motor function showed no significant improvement…
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Recorna and Starna Therapeutics Join Forces to Advance RNA Gene Editing in Lung Diseases
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Recorna (Guangzhou) Biotechnology Co., Ltd, a pioneering RNA editing therapy startup based in China, has entered into a strategic partnership with Starna Therapeutics, an mRNA specialist based in Suzhou. This alliance aims to combine their expertise to delve into the research and application of RNA gene editing drugs for lung…
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AccurEdit Therapeutics Publishes Positive Data for Gene-Edited Drug ART001 in Amyloidosis Treatment
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AccurEdit Therapeutics (Suzhou) Co., Ltd., a leading gene editing therapy specialist based in China, has published the efficacy and safety data for its drug candidate ART001, which is being investigated as a treatment for transthyroxine protein amyloidosis. ART001, the first non-viral vector in vivo gene-edited drug in China to enter…
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Roche and Genprex Initiate Phase I Trial Combining Tecentriq and Reqorsa for ES-SCLC
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Switzerland-based Roche (SWX: ROG) is advancing the evaluation of its anti-PD-L1 drug, Tecentriq (atezolizumab), in combination with Genprex’s (NASDAQ: GNPX) gene therapy, Reqorsa (quaratusugene ozeplasmid), for the treatment of extensive-stage small cell lung cancer (ES-SCLC). Genprex has initiated the dose escalation phase of a Phase I clinical trial, with the…
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ReJuKon Biopharm’s RJK002 Earns Tacit Approval for Amyotrophic Lateral Sclerosis Clinical Trial
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Shanghai-based ReJuKon Biopharm Inc. has announced that its Category 1 drug RJK002 has received tacit clinical trial approval from China’s Center for Drug Evaluation (CDE) for the treatment of amyotrophic lateral sclerosis (ALS). RJK002 is recognized as the first clinically approved adeno-associated virus (AAV) gene therapy for ALS in China…
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Profluent’s Neural Network Model Designs High-Efficiency CRISPR Gene-Editing System
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Biotechnology company Profluent has announced a significant advancement in the field of gene editing with the successful use of a neural network model to design CRISPR systems. These newly designed systems have been experimentally shown to edit genes with high efficiency and accuracy. The lead candidate from this initiative is…
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Pfizer Pauses Dosing in DMD Gene Therapy Trial Following Participant’s Sudden Death
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This week, Pfizer Inc. (NYSE: PFE) announced the unfortunate sudden death of a participant in a Phase II study for its investigational gene therapy, fordadistrogene movaparvovec, used to treat Duchenne muscular dystrophy (DMD). The patient, aged between 2 and 4 years, had received the treatment in the previous year. In…
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HuidaGene’s MECP2 Duplication Syndrome Therapy Earns Positive EMA Opinion
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HuidaGene (Shanghai) Biotechnology Co., Ltd, a biotech company based in China, has announced that it has received positive opinions from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) regarding its orphan drug filing for HG204. HG204 is an RNA editing therapy developed independently by HuidaGene,…
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Pfizer’s Gene Therapy Beqvez Wins FDA Nod for Moderate-to-Severe Hemophilia B
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The US Food and Drug Administration (FDA) has granted marketing authorization to Pfizer (NYSE: PFE) for its gene therapy Beqvez (fidanacogene elaparvovec) for the treatment of adults with moderate-to-severe hemophilia B. The one-time treatment is indicated for patients using factor IX prophylaxis therapy, those experiencing life-threatening hemorrhages, or those with…
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Bayer CEO Joins German Chancellor on China Visit, Announces New Investments
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Bayer AG (ETR: BAYN, FRA: BAYN, OTCMKTS: BAYRY), a multinational pharmaceutical and life sciences company based in Germany, recently had its CEO, Bill Anderson, accompany German Chancellor Olaf Scholz on a high-profile visit to China. In a brief interview with Xinhua, Anderson emphasized Bayer’s dedication to developing innovative drugs and…
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Shanghai’s 9th People’s Hospital Launches Groundbreaking Gene Therapy Trial for Congenital Deafness
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In a medical first, Shanghai’s 9th People’s Hospital has initiated the world’s inaugural clinical trial for a gene-editing therapy targeting congenital deafness attributed to otoferlin (OTOF) gene mutations. The therapy, developed by HuidaGene Therapeutics Co., Ltd, represents a significant advance in the treatment of auditory neuropathy, a leading cause of…
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China’s CNIPA Upholds Patent for Nobel-Winning CRISPR/Cas9 Gene Editing Technology
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The China National Intellectual Property Administration (CNIPA) has upheld a key patent surrounding the CRISPR/Cas9 gene editing technology, rejecting an invalidation challenge. The decision was announced by ERS Genomics Ltd, an Ireland-based company founded by Dr. Emmanuelle Charpentier, the Nobel prize-winning co-inventor of the technology, to manage and license access…
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Regenxbio Reports Stable Visual Acuity Improvements with AbbVie’s Gene Therapy in Wet AMD Trial
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Regenxbio (NASDAQ: RGNX), the development partner for AbbVie’s (NYSE: ABBV) gene therapy ABBV-RGX-314, has announced results from a Phase I/IIa trial focused on wet age-related macular degeneration (wAMD). This announcement complements the preliminary results shared earlier in the year. The 2-year data from the trial indicate that a single administration…
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Shanghai Medicilon Forms Strategic Partnerships with Binhui Biopharmaceutical and Innoforce Pharmaceuticals
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Shanghai Medicilon Biomedical Co., Ltd (SHA: 688202), a prominent Chinese biotech firm, has entered into separate partnerships with domestic companies Binhui Biopharmaceutical and Innoforce Pharmaceuticals Co., Ltd. In collaboration with Binhui, Medicilon aims to explore innovative areas including oncolytic viruses, nucleic acid drugs, and protein therapeutics. Meanwhile, the partnership with…
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Roche Invests EUR 90 Million in New Gene Therapy Center in Germany
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Swiss pharmaceutical giant Roche (SWX: RO) has inaugurated a new gene therapy development center in Germany, following an investment of EUR 90 million (USD 97.4 million). The expansive 2,500 square meter facility is designed to specialize in the production of gene vectors for clinical trials across various therapeutic areas, including…
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NeuExcell Therapeutics Doses First Patient in Groundbreaking Clinical Trial for NeuroD1 Gene Therapy
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NeuExcell Therapeutics, a Shanghai-based developer of in vivo neural regenerative therapies, has announced the first patient dosing in a clinical trial for its first-in-class (FIC) NeuroD1 gene therapy product, NXL-004, targeting malignant glioma. This marks NXL-004 as the world’s first gene therapy of its kind to enter the clinical setting.…
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VectorBuilder Partners with Minaris Regenerative Medicine to Enhance Gene Therapy Solutions
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VectorBuilder, a specialist in genetic engineering based in China, has entered into a strategic partnership with Minaris Regenerative Medicine, a cell and gene therapy (CGT) Contract Development and Manufacturing Organization (CDMO) and a subsidiary of Japan-headquartered Showa Denko Materials Co., Ltd. This collaboration aims to integrate VectorBuilder’s superior R&D and…
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Novartis Gene Therapy Zolgensma Shows Continued Benefits in Broader SMA Patient Group
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Swiss pharmaceutical giant Novartis (NYSE: NVS) has presented Phase III data this week, demonstrating that the clinical benefits of its gene therapy Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy (SMA) extend beyond patients up to 2 years of age. The new study has shown similar benefits in older children, including…
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CANbridge Pharmaceuticals Partners with Peking Union Medical College to Advance Rare Disease Research
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CANbridge Pharmaceuticals Inc. (HKG: 1228), a specialist in rare diseases based in China, has announced a partnership with Peking Union Medical College Hospital to collaborate on an innovative study spanning drug discovery to clinical translation for rare diseases. The partnership aims to establish a rare disease innovation development platform focused…
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Shenzhen Eureka Biotechnology Secures RMB 300 Million in Series B+ Funding for CGT Expansion
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Shenzhen Eureka Biotechnology Co., Ltd., a specialist in cell and gene therapy (CGT) based in China, has successfully raised over RMB 300 million (approximately USD 41.7 million) in a Series B+ financing round. The funding was led by Yuexiu Capital, with notable participation from Fortune Capital, Guangdong Technology Financial Group,…
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Neurophth Biotechnology Completes Enrollment and Dosing in US Clinical Study for ND4-LHON Gene Therapy
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Neurophth Biotechnology Ltd, a leading gene therapy specialist based in China, has announced the completion of patient enrollment and dosing in a Phase I/II clinical study for its investigational drug NR082 (rAAV2-ND4; NFS-01) in the United States. The study targets ND4-mediated Leber’s hereditary optic neuropathy (ND4-LHON). The single-arm, multi-center Phase…
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ChinaGene Secures CDE Approval for ZVS101e Gene Therapy Targeting Retinitis Pigmentosa
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Beijing-based ChinaGene Technology Co., Ltd. has announced that it has received tacit clinical approval from the Center for Drug Evaluation (CDE) for its ophthalmic gene therapy ZVS101e, designed to treat retinitis pigmentosa (RP) patients with the RHO-R135W (RHO c.403C>T) mutation. This follows a tacit trial approval granted in the United…
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J&J’s Gene Therapy for X-linked Retinitis Pigmentosa Receives CDE Nod for Breakthrough Designation
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The Center for Drug Evaluation (CDE) website has indicated that Johnson & Johnson (J&J, NYSE: JNJ)’s AAV5-hRKp.RPGR (JNJ-5340, botaretigene sparoparvovec) intraocular injectable solution is on track to receive breakthrough therapy designation (BTD). This product, an adeno-associated virus vector (AAV) based gene therapy co-developed with MeiraGTx Holdings plc, is aimed at…
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Astellas Forges Partnership with Mass General Brigham to Boost Oncology and Rare Disease Research
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Astellas Pharma Inc. (TYO: 4503), a leading Japanese pharmaceutical company, has announced a strategic partnership with Mass General Brigham, a prominent academic institution in the United States. This collaboration aims to drive scientific and clinical advancements in critical areas, including oncology, rare diseases, and cell and gene therapy. Astellas will…
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Eli Lilly’s Subsidiary Akouos Reports Positive Preliminary Results for Gene Therapy in Hearing Loss
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Eli Lilly (NYSE: LLY) has announced preliminary results from a Phase I/II study conducted by its subsidiary Akouos for gene therapy AK-OTOF, which is being investigated for the treatment of sensorineural hearing loss due to mutations in the otoferlin gene (OTOF). The press release reported that the first participant in…
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GSK In-Licenses Elegen’s Cell-Free DNA Tech to Advance Next-Gen Gene Therapies and Vaccines
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GlaxoSmithKline (GSK; NYSE: GSK), a UK-based pharmaceutical giant, has this week in-licensed US partner Elegen’s cell-free DNA manufacturing technology to bolster the potential development of next-generation mRNA, cell, and viral gene therapies, as well as RNA-based vaccines. Elegen will receive upfront fees, purchase commitments, potential equity investment, and milestone payments…
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Lingyi Biotechnology’s Gene Therapy Candidate LY-M001 Gets FDA Green Light for Gaucher’s Disease
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Lingyi Biotechnology Co., Ltd, a China-based gene therapy developer, has secured clinical trial approval from the US Food and Drug Administration (FDA) for its proprietary AAV gene therapy candidate, LY-M001, which is being developed to treat type I or III Gaucher’s disease. LY-M001 utilizes a recombinant adeno-associated virus (rAAV) as…
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Guangdong Aims to Boost Biomedicine Industry with Incentives and Strategic Development
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The Guangdong Provincial Development and Reform Commission has unveiled an ambitious set of measures aimed at fostering high-quality development in the biomedicine industry. The initiative seeks to propel Guangzhou’s biopharmaceutical sector to overcome challenges, enhance metropolitan coordination, and spearhead industry growth in the Greater Bay Area (GBA). The program extends…
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FDA Approves Casgevy, the First CRISPR-Based Gene Editing Therapy for β-Thalassemia
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Casgevy (exagamglogene autotemcel), the pioneering gene editing therapy based on CRISPR technology, has received approval from the US Food and Drug Administration (FDA) for the treatment of patients aged 12 and above with transfusion-dependent β-Thalassemia (TDT). This marks a significant milestone as Casgevy becomes the world’s first CRISPR-based gene editing…
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Regenxbio’s One-Time Gene Therapy for Wet AMD Shows Promise in Phase II Study
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Gene therapy specialist Regenxbio (NASDAQ: RGNX) has announced preliminary results from a Phase II study for ABBV-RGX-314, a VEGF pathway inhibitor developed in partnership with AbbVie (NYSE: ABBV) for the treatment of wet age-related macular degeneration (wet AMD). The therapy is designed to be administered as a one-time injection, aiming…
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Bayer’s Asklepios BioPharmaceutical Launches Phase II Trial for AAV Therapy in Heart Failure
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Bayer’s (ETR: BAYN) gene therapy subsidiary, Asklepios BioPharmaceutical, has initiated patient recruitment for a Phase II clinical trial of its adeno-associated virus (AAV) therapy AB-1002 targeting congestive heart failure (CHF). This placebo-controlled study aims to assess the safety and efficacy of intracoronary infusion in 90 to 150 adults with non-ischemic…
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Shanghai BDgene Technology’s Thalassemia Gene Therapy BD211 Clears NMPA for Clinical Trials
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Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has received clinical trial approval from the National Medical Products Administration (NMPA) for its therapy BD211. This gene-modified autologous hematopoietic stem cell therapy is designed for the treatment of thalassemia. In a preliminary study, BD211 demonstrated safety and efficacy in…
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Belief BioMed Group Initiates Clinical Study for Hemophilia A Gene Therapy BBM-H803 in China
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Belief BioMed Group, a Shanghai-based gene therapy specialist, has announced the first subject dosing in a regulatory clinical study for its in-house developed adeno-associated virus (AAV) gene therapy, BBM-H803, for the treatment of hemophilia A in China. This marks a significant step forward in addressing the unmet medical need for…
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China’s CDE Releases Guidelines for CGT Clinical Trials Communication
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The Center for Drug Evaluation (CDE) in China has released the “Technical Guiding Principles for Cell and Gene Therapy (CGT) Product Clinical Communication and Exchange,” addressing the unique considerations of personalized and specific CGT products. Applicants are advised to focus on critical factors such as product characteristics, clinical positioning, target…
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Janssen Acquires Rights to Gene Therapy Botaretigene Sparoparvovec from MeiraGTx for XLRP Treatment
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Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson (J&J, NYSE: JNJ), announced last week its acquisition of development, manufacturing, and commercialization rights for the gene therapy botaretigene sparoparvovec from MeiraGTx Holdings (NASDAQ: MGTX) for the treatment of X-linked retinitis pigmentosa (XLRP). Janssen will pay an upfront fee of USD 65…
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Takeda Ends Gene Therapy Collaboration with JCR Pharmaceuticals, Returns IP
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Takeda Pharmaceuticals Company Limited (TYO: 4502) has announced the termination of a 2022 collaboration agreement with fellow Japanese firm JCR Pharmaceuticals Co., Ltd. (TYO: 4552) for the development of gene therapies. As a result of the termination, Takeda will return all research outcomes and patents to JCR. This strategic decision…
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HuidaGene Secures Rare Pediatric Disease Designation for CRISPR Therapy HG302 Targeting DMD
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HuidaGene Therapeutics, based in China, has announced that it has received Rare Pediatric Disease Designation (RPDD) from the U.S. FDA for its CRISPR-Cas12 DNA editing therapy, HG302. This designation highlights the therapy’s potential to treat Duchenne muscular dystrophy (DMD), a severe neuromuscular genetic disorder. HG302 employs high fidelity Cas12 (hfCas12Max)…
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China’s State Council Unveils Comprehensive Plan to Boost Beijing’s Healthcare Sector
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The State Council has unveiled a comprehensive “Work Plan” aimed at enhancing Beijing’s service industry capabilities, with a particular focus on healthcare and medical services. Titled “Work Plan Supporting the Construction of Comprehensive Demonstration Zones to Deepen National Service Industry Openness in Beijing,” the document outlines several key proposals that…
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AstraZeneca to Open R&D Center in Hong Kong, Focusing on Cell and Gene Therapies
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UK pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) has concluded a roughly 12-month discussion period with local authorities and is proceeding to establish a research and development (R&D) center in Hong Kong, marking a significant step as it becomes the first leading global Big Pharma to do so in the city.…
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Vertex Pharmaceuticals and CRISPR Therapeutics Receive Conditional Approval for CRISPR Gene-Editing Therapy
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Vertex Pharmaceuticals (NASDAQ: VRTX) and CRISPR Therapeutics (NASDAQ: CRSP) have received conditional approval from the UK Medicines and Healthcare products Regulatory Agency (MHRA) for their jointly developed therapy, Casgevy (exagamglogene autotemcel), a treatment for sickle cell disease (SCD) with recurrent vaso-occlusive crises (VOCs) and for transfusion-dependent beta thalassemia (TDT) in…
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Clinical Research on rAAV Gene Drug ZS805 for Fabry Disease Launched by Zhishan Weixin
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Zhishan Weixin Biotechnology Co., Ltd, a China-based biotech company, has announced the launch of the first clinical research project in China focused on a recombinant adeno-associated virus (rAAV) genetic therapy for the treatment of Fabry disease. The study will evaluate the adeno-associated virus vector ZS805 as a potential treatment across…
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Biosmelt Pharma Partners with Shenzhen Eureka Biotechnology for Cell and Gene Therapy Advancements
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Shanghai-based cell and gene therapy (CGT) specialist, Biosmelt Pharma, has entered into a partnership with Shenzhen Eureka Biotechnology Co., Ltd, a domestic CGT-focused Contract Development and Manufacturing Organization (CDMO) service provider. Combining Expertise for Large-Scale Virus Vector ProductionThe collaboration aims to capitalize on Biosmelt’s GMP grade lentivirus vector suspension production…
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Suzhou Porton Biologics Partners with BioMap to Enhance AAV Vector Design in Gene Therapy
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Suzhou Porton Biologics Ltd, a China-based Contract Development and Manufacturing Organization (CDMO), has entered into a strategic partnership with US biotech company BioMap, an AI-based drug discovery platform co-founded by Baidu’s CEO Robin Li and Baidu Ventures CEO Wei Liu. This collaboration aims to develop an AAV assembly efficiency model…
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Bayer and Shanghai Pharmaceuticals Launch Co.Lab to Incubate Life Sciences Innovation in China
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Bayer, the Germany-headquartered multinational pharmaceutical and life sciences company (ETR: BAYN), has entered into a partnership with Shanghai Pharmaceuticals, a leading China-based pharmaceutical distribution giant (SPH; HKG: 2607; SHA: 601607), to co-construct a new life sciences incubator named Co.Lab. This collaboration is designed to foster cutting-edge innovation in cell and…
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AstraZeneca Partners with Cellectis for Next-Generation Cell and Gene Therapies
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UK pharmaceutical giant AstraZeneca (AZ; NASDAQ: AZN) has announced a strategic partnership with Cellectis (EPA: ALCLS; NASDAQ: CLLS) to develop next-generation therapeutics, focusing on areas of high unmet need such as oncology, immunology, and rare diseases. This collaboration aims to leverage Cellectis’s gene editing technologies and manufacturing capabilities to advance…
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Bio-Techne Corporation Reports 2% YOY Revenue Growth in Q1 2024 Fiscal Report
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US-based life sciences service provider Bio-Techne Corporation (NASDAQ: TECH) has released its Q1 report for the fiscal year 2024, which ended on September 30, 2023. The company reported a 2% year-on-year (YOY) increase in revenues, reaching USD 276.9 million. Segment Performance and Revenue BreakdownBio-Techne’s Protein Sciences segment contributed USD 204.7…
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Roche’s Gene Therapy Elevidys Misses Primary Endpoint in DMD Trial
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Switzerland-headquartered pharmaceutical giant Roche (SWX: ROG) has announced that a Phase III trial for its gene transfer therapy Elevidys (delandistrogene moxeparvovec) did not meet its primary endpoint in Duchenne muscular dystrophy (DMD) ambulatory patients aged 4 to 7 years. The trial aimed to measure the change from baseline in motor…
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Novartis Seeks Access to Takeda’s Business Records Over Confidentiality Concerns
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Swiss pharmaceutical giant Novartis (NYSE: NVS) filed a complaint with a US court last week, seeking discovery to gain access to certain business records of Japan-based Takeda (TYO: 4502). Novartis aims to determine whether Takeda has potentially benefited from confidential documents that may have been inappropriately transferred by a former…
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Strategic Alliance Formed by uBriGene, Fraserna, and Virogin for Cell and Gene Therapy Services
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In a significant move to bolster the cell and gene therapy (CGT) sector, China-based biotechnology firms uBriGene, Fraserna, and Virogin have entered into a strategic partnership agreement. While the financial details of the agreement remain undisclosed, the collaboration is poised to integrate the global resources of the three companies, offering…
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Skyline Therapeutics Gains NMPA Approval for SKG0106 in Neovascular AMD Treatment
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Skyline Therapeutics has announced that it has received clinical trial approval from the National Medical Products Administration (NMPA) for its innovative drug SKG0106, an intraocular injection solution for the treatment of neovascular age-related macular degeneration (nAMD). Innovative Gene Therapy MechanismSKG0106 is a recombinant adeno-associated virus (rAAV) gene therapy drug, utilizing…
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Accuredit Therapeutics Initiates First Dosing of Gene Therapy ART001 for ATTR
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China-based gene therapy start-up Accuredit Therapeutics (Suzhou) Co., Ltd has announced the first dosing of its investigational drug ART001 in seven subjects with transthyretin amyloidosis (ATTR), as part of an investigator-initiated trial (ITT). The drug candidate has demonstrated good safety and achieved a clinically significant decrease in transthyroxine (TTR) protein…
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Belief BioMed Partners with Takeda for BBM-H901 Commercialization in Asia
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Shanghai-based gene therapy specialist Belief BioMed Group (BBM) has announced a strategic partnership with Japan’s Takeda Pharmaceutical Company (TYO: 4502), granting Takeda commercialization rights for its investigational product, BBM-H901. This agreement encompasses the territories of mainland China, Hong Kong, and Macau. Financial details of the partnership have not been disclosed.…
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Gritgen Therapeutics Commences Operations of Commercial GMP Facilities in Suzhou
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Suzhou-based gene therapy specialist, Gritgen Therapeutics Co., Ltd, has announced the validation completion and initiation of operations at its commercial Good Manufacturing Practice (GMP) facilities located in the Suzhou Industrial Park. This development marks a significant step forward for the company in advancing its capabilities in gene therapy production. Facility…
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Shanghai BDgene’s BD112 Earns Orphan Drug Designation from US FDA for Huntington’s Disease
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Shanghai BDgene Technology Co., Ltd, a specialist in gene therapy, has announced that its drug candidate BD112, which utilizes virus-like particle (VLP) delivery technology for in vivo gene editing, has received orphan drug designation (ODD) from the US Food and Drug Administration (FDA). This follows a previous ODD granted by…
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Kyowa Hakko Kirin to Acquire Orchard Therapeutics for $477.6 Million
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Japan-based Kyowa Hakko Kirin is poised to acquire UK-headquartered Orchard Therapeutics plc for a total consideration of USD 477.6 million. The deal comprises USD 387.4 million in cash and USD 90.2 million in Contingent Value Rights (CVRs), with the transaction expected to close in the first quarter of 2024. Acquisition…
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Regeneron Pharmaceuticals Acquires Decibel Therapeutics to Advance Hearing Loss Treatments
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Regeneron Pharmaceuticals (NASDAQ: REGN) has completed the acquisition of Decibel Therapeutics, a developer of gene therapies for hearing loss, following an agreement reached last month. This strategic move expands Regeneron’s portfolio in the field of genetic therapies and underscores the company’s commitment to addressing hearing loss conditions. Decibel’s Pipeline and…
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Suzhou Novoprotein and Shanghai Model Organisms Center Ink Global Partnership
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Suzhou Novoprotein Scientific Co., Ltd, (SHA: 688137) a leading provider of protein technology and application solutions in China, has entered into a strategic partnership with Shanghai Model Organisms Center, Inc. (SHA: 688265). The collaboration aims to combine the companies’ expertise on a global scale, focusing on technology and commerce. Novoprotein…
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Suzhou Porton Biologics Partners with BRL Medicine to Advance Gene and Cell Therapy Pipeline
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China-based Contract Development and Manufacturing Organization (CDMO) Suzhou Porton Biologics Ltd has entered into a strategic partnership with compatriot firm BRL Medicine Inc. The collaboration aims to deepen their involvement in the gene and cell therapy (GCT) pipeline, enhance clinical studies, and expedite the commercialization of novel therapies. BRL Medicine’s…
