FDA approves first-ever gene therapy to be directly administered to the brain
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.
Cell & gene therapies
Abzena’s advanced drug development showcased at CPHI Milan
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.
Aldevron’s Anu Codaty on passion, leadership, and innovation
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.
Phastar's Vicky Marriott: Empowering women in STEM with empathy
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.
Women in STEM: Gitte Barknowitz’s journey to leading global initiatives
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 on leading regulatory affairs at CTMC: A journey from mAbs to cellular therapies
Jane Koo, head of regulatory affairs at CTMC, plays an important role in accelerating the development and commercialization of innovative cellular therapies.
Women in Science - SCIEX Rebekah Sayers' on encouraging curiosity
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.
Quotient Sciences expands with mRNA Focus at CPHI 2024
During the show, Quotient Sciences CEO Thierry Van Nieuwenhove will share insights on how the company is adapting to new FDA regulations.
FDA designations boost AI-driven cell therapy approach for Duchenne muscular dystrophy
The FDA has granted Orphan Drug and Rare Pediatric Disease Designations to Somite’s cell replacement therapy for the treatment of Duchenne muscular dystrophy.
Women in Science - Emmanuelle Cameau on growing stronger from challenges
Emmanuelle Cameau is strategic technology partnership leader at Cytiva.
Neuromuscular diseases in crosshairs in Forge Biologics gene therapy collaboration
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.
Women in Science - Beate Mueller on the privilege of bringing solutions closer to patients
Beate Mueller-Tiemann is chief technology officer at Cytiva.
Candid Therapeutics launches with $370m to tackle autoimmunity with T-cell engagers
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 secures second patent for AI-powered oligonucleotide engineering
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.
Innovative nanoparticle method could completely change therapeutics
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 gains accelerated FDA approval for rare liver disease treatment
Gilead wins on new company acquisition with accelerated approval for autoimmune liver disease drug
Adaptimmune’s Tecelra becomes the first FDA-approved cell therapy for a solid tumor
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 announces discontinuation of DMD gene therapy program and lets go of 150 staff
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 raises $90 million Series A to bring RNA editing therapies to the clinic
AIRNA plans to bring RNA editing therapies to the clinic starting with a treatment for the genetic disease alpha-1 antitrypsin deficiency.
Women in Science - Tehmina Masud - the importance of growing outside your comfort zone
A book, a computer and some sage words from her physicist father set Tehmina on her path to a successful career in science.
Rona Therapeutics raises $35M in series A+ to advance metabolic siRNA therapeutics
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.
Pluri’s CDMO arm takes on ALS and diabetes in cell therapy deal
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 announces positive early results for Angelman syndrome therapy
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.
Top 5 CDMOs driving advanced therapies to market amid rising demand and challenges
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...
Lexeo’s gene therapy shows promising phase 1/2 results for Friedreich's ataxia cardiomyopathy
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.
IDT's new 25,000 sq. ft. synthetic biology facility doubles production capacity
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 new EU ultra-cold facility to boost advanced therapies development
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.
ReciBioPharm’s Edita Botonjic-Sehic on analytics, AI, and continuous RNA manufacturing
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 selected to manufacture clinical grade AAV9-CSA vector to treat Cockayne syndrome
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...
Ones to watch - five upcoming companies on the CAR-T scene
CAR-T therapies have big potential to take on blood cancer but also have major drawbacks.
ReciBioPharm’s Edita Botonjic-Sehic, on analytics, AI, and continuous RNA manufacturing
RNA therapeutics have emerged as a transformative approach to treating many conditions, but the industry still typically relies on inefficient batch manufacturing methods.
UK, Denmark set up payment models for Hemgenix hemophilia B gene therapy
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.
Disappointment for Rocket as FDA delays approval of gene therapy further
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.
PolTREG’s regulatory T cell therapy shows promise in type-1 diabetes
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.
Mainz Biomed CEO discusses stool test for colorectal cancer vs Galleri blood test
Catching cancer early can save lives because it can be treated before it spreads in the body.
Maryam Abdul-Kareem - to women I say 'have confidence and don't allow people to discount you'
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.
CAR-T therapy achieves lupus remission breakthrough
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 EU ultra-cold facility to boost advanced therapies development
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.
AI-fueled protein design startup spun out from Cyrus Biotechnology
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 and STADA to co-develop biosimilars for bone disease treatments
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.
Women in Science: When you follow your passiojn for science - your capabilities will shine through
Daria Donati is chief scientific officer for Genomic Medicine at Cytiva.
Inbar Friedrich Ben-Nun on curiosity, resiliency, and the discovery of what's next.
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.
ASCO: AffyImmune builds up foundations of autologous CAR T cell therapy
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’ CAR-T cell therapy shows promise against autoimmune diseases
Kyverna Therapeutics has unveiled compelling new data for its CAR T-cell therapy, KYV-101, which may revolutionize the treatment of autoimmune diseases.
Three Indian CDMOs ready to seize the BIOSECURE Act opportunity
In the wake of escalating tensions between the United States and China, the Biden Administration is intensifying its strategic focus on biosecurity.
Could DefiniGEN and Atelerix’s new collaboration revolutionize cell model shipping?
In what could be a significant leap for scientific research and biopharmaceuticals, DefiniGEN and Atelerix have unveiled a new collaboration that allows in vitro cell models to be shipped internationally without freezing or cryopreservation.
Bionova Scientific announces plans for new Plasmid DNA GMP facility
Bionova Scientific, an Asahi Kasei Group company and full-service biologics CDMO, has announced plans to expand into plasmid DNA (pDNA) production.
BIO International 2024 to focus on partnerships amid new BIOSECURE bill
As the BIO International 2024 trade show approaches, industry insiders anticipate a significant focus on partnership discussions, driven by the pressing financial needs of biotech companies rather than the new BIOSECURE act.
Cellular Origins and CGT Catapult partner to automate cell and gene therapy manufacturing
Cellular Origins and the Cell and Gene Therapy Catapult (CGT Catapult) have announced a new collaboration to demonstrate universal automation of CGT manufacturing.
How EXO Biologics plans to revolutionize exosome therapy with €16m cash injection
EXO Biologics, a clinical-stage biotech specializing in exosome-based therapies, recently announced the successful raising of €16 million in Series A funding.