News & Analysis on the Clinical Development and Manufacture of Large Molecule Drugs
The US gene therapy company Regenxbio and the Japanese firm Nippon Shinyaku have joined forces to boost the development and commercialization of Regenxbio’s gene therapies RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II) and RGX-111 for...
Following a positive meeting with the FDA, BrainChild is getting ready to initiate a pivotal registration trial with its lead cell therapy candidate.
Mesoblast has received FDA approval for Ryoncil, becoming the first and only cell therapy of its kind to be available in the US.
The merger of Nvelop Therapeutics and Chroma Medicine brings together a combination of epigenetic editing and in vivo delivery expertise to develop new genetic medicines.
The sickle cell gene therapies developed by Bluebird Bio and Vertex will be the first to be offered under the new scheme.
The acquisition will grant Roche full ownership over a pipeline of allogeneic CAR-T therapy candidates, including two oncology programs it had previously in-licensed.
PTC Therapeutics has received FDA approval for a gene therapy to treat AADC deficiency, a rare genetic disorder that impairs the normal function of neurons.
At CPHI Milan 2024, Abzena showcased its latest innovations in cell line development and ADC manufacturing, featuring the advanced AbZelectPRO platform and ThioBridge linker technology.
Anu Codaty, VP of global marketing and strategy at Aldevron, is blazing trails in biotech, inspired by a family legacy in medicine and driven to create life-changing therapies.
Vicky Marriott’s journey from enjoying school math to heading a global team of statisticians at Phastar reveals her belief in the power of empathy and collaboration.
From exploring the wonders of plants as a curious teen to leading global initiatives at SCIEX, Gitte Barknowitz has carved out an exciting and unconventional career in science.
Jane Koo, head of regulatory affairs at CTMC, plays an important role in accelerating the development and commercialization of innovative cellular therapies.
Rebekah's father passed on his passion for science and problem solving to her and much of her childhood was spent doing fun hands-on activities from taking apart household mechanics to building rockets.
During the show, Quotient Sciences CEO Thierry Van Nieuwenhove will share insights on how the company is adapting to new FDA regulations.
The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the treatment of Duchenne muscular dystrophy.
Emmanuelle Cameau is strategic technology partnership leader at Cytiva.
Forge Biologics will help with the manufacturing of adeno-associated viruses (AAVs) in a collaboration with the non-profit Muscular Dystrophy Association (MDA) to develop gene therapies for neuromuscular diseases.
Beate Mueller-Tiemann is chief technology officer at Cytiva.
The San Diego-based biotech company Candid Therapeutics has launched with a huge $370 million with the aim to develop T-cell engager therapies for autoimmune diseases.
Creyon Bio Inc., a clinical-stage biotechnology company specializing in AI and machine learning-enabled oligonucleotide-based medicines (OBMs), recently announced the issuance of its second US patent.
In a significant leap forward for therapeutic science, researchers at Cornell University have developed a pioneering method to 'cloak' proteins, enabling their efficient delivery into living cells.
Gilead wins on new company acquisition with accelerated approval for autoimmune liver disease drug
The FDA has granted accelerated approval to Adaptimmune’s Tecelra, a T cell therapy for the treatment of synovial sarcoma when other lines of treatment do not work.
Pfizer will let go of 150 employees at its Sanford, US site that were involved in the development of a gene therapy for Duchenne muscular dystrophy that recently failed in a phase 3 clinical trial.
AIRNA plans to bring RNA editing therapies to the clinic starting with a treatment for the genetic disease alpha-1 antitrypsin deficiency.
A book, a computer and some sage words from her physicist father set Tehmina on her path to a successful career in science.
The fundraising will support the development of the company’s technology platform for silencing RNA (siRNA), which has the potential to enable dosing patients every six months to treat a wide range of conditions.
The contract development and manufacturing organization (CDMO) arm of the Israeli company Pluri will deploy automated and 3D cell expansion technology to produce cell therapies developed by compatriot firm Kadimastem.
Ionis Pharmaceuticals antisense therapy for Angelman syndrome achieved good early safety and efficacy results and the company now plans to advance the therapy to phase 3.
Contract development and manufacturing organizations (CDMOs) perform vital services for the biopharmaceutical industry – a role that is growing as cell and gene therapies gain momentum. Here are five of the top CDMOs bringing advanced therapies toward...
A gene therapy being developed by Lexeo Therapeutics to treat cardiomyopathy caused by Friedreich ataxia, a rare neurodegenerative genetic disease, has achieved good interim results in a phase 1/2 trial.
Integrated DNA Technologies (IDT) is doubling down on its commitment to innovation with the launch of a new 25,000-square-foot synthetic biology facility in Coralville, Iowa.
Thermo Fisher’s cutting-edge ultra-cold facility in Bleiswijk, Netherlands, aims to accelerate advanced therapies development by offering comprehensive cold and ultra-cold services.
RNA therapeutics have emerged as a transformative approach to treating many conditions, but the industry still typically relies on inefficient batch manufacturing methods.
Andelyn Biosciences, a leading cell and gene therapy Contract Development and Manufacturing Organization (CDMO), has been chosen by UMass Chan Medical School to manufacture clinical-grade AAV9-CSA vector using its suspension AAV Curator Platform to...
CAR-T therapies have big potential to take on blood cancer but also have major drawbacks.
RNA therapeutics have emerged as a transformative approach to treating many conditions, but the industry still typically relies on inefficient batch manufacturing methods.
Hot on the heels of last year’s EU approval of Hemgenix, the first gene therapy for the blood disorder hemophilia B, the UK and Denmark are the latest European nations to take steps to pay for the treatment.
The US FDA has delayed approval of Rocket Pharmaceuticals’ gene therapy for treatment of a rare immune condition known as leukocyte adhesion deficiency, citing a need for more information about the manufacturing process.
Results from a phase 1/2 clinical trial revealed that PolTREG’s regulatory T cell (Treg) therapy, PTG-007, was able to restore insulin secretion in children with type-1 diabetes.
Catching cancer early can save lives because it can be treated before it spreads in the body.
Described by colleagues as an exceptional leader and champion of equity and diversity, this week's woman in science is Maryam Abdul-Kareem, the general counsel of Arcellx.
New data from a phase 1 clinical trial shows complete remission in 92% of lupus patients treated with a compound CAR (cCAR) T cell therapy
Thermo Fisher’s new state-of-the-art ultra-cold facility in Bleiswijk, Netherlands, aims to accelerate advanced therapies development by offering a comprehensive range of cold and ultra-cold services.
The Seattle-based company Cyrus Biotechnology has spun out Levitate Bio to offer artificial intelligence (AI)-based protein design software to customers across the biopharma space.
Alvotech of Iceland and STADA Arzneimittel of Germany have extended an existing partnership to co-develop a clinical-stage biosimilar for Prolia and Xgeva, Amgen’s therapies for bone conditions based on the antibody drug denosumab.
Daria Donati is chief scientific officer for Genomic Medicine at Cytiva.
Director of cell and gene therapy R&D at Lonza, this week's inspiring female is Inbar who we spoke to about her passion for discovering the future and mentor support sparked her early interest in biology and informed her journey of resiliency.
In spite of their potential to tackle cancer in the long term, CAR-T cell therapies face challenges in terms of pricing, manufacturing and being limited to blood cancer.
Kyverna Therapeutics has unveiled compelling new data for its CAR T-cell therapy, KYV-101, which may revolutionize the treatment of autoimmune diseases.