Are haemophilia gene therapies truly “one and done”?
Pharmaceutical Technology
NOVEMBER 20, 2023
The value of haemophilia gene therapies like Roctavian and Hemgenix hinge on long-term efficacy and innovative payment structures play a key role.
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Ajinomoto spices up its biopharma business with gene therapy deal
Bio Pharma Dive
NOVEMBER 13, 2023
Forge Biologics operates a contract manufacturing business as well as develops its own gene therapies, which appears to have attracted the Japan-based food and biotechnology company.
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Why are haemophilia gene therapies so expensive?
Pharmaceutical Technology
OCTOBER 11, 2023
Biomarin and CSL Behring’s haemophilia gene therapies have boosted innovation in the field, but assessing their cost effectiveness is less than straightforward.
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Experts Believe Gene Therapy Offers Potential to Impact Gaucher Disease Market
Pharmaceutical Technology
NOVEMBER 21, 2023
According to several key opinion leaders (KOLs) interviewed by GlobalData, pipeline gene therapy holds the greatest potential to transform the Gaucher disease landscape
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Making the Case for Case Management for Cell and Gene Therapies
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As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements.
Orchard sells to Kyowa Kirin in gene therapy buyout
Bio Pharma Dive
OCTOBER 5, 2023
Kyowa will pay nearly $400 million to acquire Orchard, which sells the gene therapy Libmeldy in Europe but has struggled to find paths to market for other experimental treatments.
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How gene therapies can transform sickle cell disease treatment
Pharmaceutical Technology
OCTOBER 26, 2023
The potential for using gene therapies to treat sickle cell disease is high, but their durability and high price point invite scrutiny.
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Spark Therapeutics partners with SpliceBio for gene therapy
Pharmaceutical Technology
OCTOBER 17, 2023
SpliceBio will license Spark Therapeutics’ propriety protein splicing platform to develop a gene therapy for renal disease.
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Forge Biologics and Labcorp partner for gene therapies development
Pharmaceutical Technology
MAY 4, 2023
Forge Biologics and global life sciences company Labcorp have announced a strategic adeno-associated virus (AAV) gene therapy development and manufacturing collaboration. This will improve accessibility to services for AAV-mediated gene therapy programmes.
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Cyagen and Neurophth partner to develop gene therapy vectors
Pharmaceutical Technology
NOVEMBER 8, 2022
Cyagen and Neurophth Therapeutics have entered a strategic partnership to jointly develop next-generation AAV gene therapy vectors for specific kinds of genetic ophthalmic ailments. Additionally, Neurophth will oversee the clinical trials and marketing of gene therapy products developed leveraging the new AAV capsids of Cyagen.
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Avrobio to sell cystinosis gene therapy to Novartis for $87.5m
Pharmaceutical Technology
MAY 23, 2023
Avrobio has announced a deal to sell its investigational haematopoietic stem cell (HSC) gene therapy programme , designed to treat cystinosis, to Novartis in an all-cash deal valued at $87.5m. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Beacon Therapeutics enters gene therapy arena with $120m launch
Pharmaceutical Technology
JUNE 13, 2023
Beacon Therapeutics has kickstarted its entry into the gene therapy field with a $120m Series A financing. Amongst it was AGTC’s lead clinical candidate, AFTC-501, an adeno-associated virus (AAV) gene therapy for XLRP. Beacon Therapeutics is the third gene therapy company Syncona has launched.
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Robotics: Transforming cell and gene therapy manufacturing
Pharmaceutical Technology
AUGUST 16, 2023
Several companies are developing robotics to automate cell and gene therapy manufacturing, but some challenges persist.
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Gene therapy startup Vedere to close two years after launch
Bio Pharma Dive
APRIL 2, 2023
The startup, a successor to an eye gene therapy biotech that Novartis bought in 2020, will wind down after preclinical experiments didn’t meet the bar set by its leaders.
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Laurus Labs and IIT Kanpur partner for new gene therapy products
Pharmaceutical Technology
JUNE 19, 2023
Indian pharmaceutical company Laurus Labs has signed a memorandum of agreement (MoA) with the Indian Institute of Technology, Kanpur (IIT Kanpur) to bring new gene therapy products to the market. Laurus Labs CEO Satyanarayana Chava stated: “This collaboration exhibits our commitment towards the cell and gene therapy (CGT) space.
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CSL doses first patient with haemophilia B gene therapy Hemgenix
Pharmaceutical Technology
JUNE 21, 2023
CSL announced the first patient with haemophilia B has been treated with its recently approved gene therapy Hemgenix (etranacogene dezaparvovec). Hemgenix is the first and only gene therapy approved for haemophilia B. per dose, it is the most expensive single-use gene therapy in the US.
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Voyager licenses gene therapy tools to Novartis
Bio Pharma Dive
MARCH 6, 2023
The Swiss drugmaker's decision is the latest in a string of partnership announcements for the gene therapy maker, which aims to bounce back from past research failures.
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Sarepta offers early look at closely watched gene therapy launch
Bio Pharma Dive
AUGUST 3, 2023
One patient received its Duchenne gene therapy, Elevidys, this week and a handful more could follow shortly, surprising some analysts and raising near-term sales expectations.
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4D Molecular’s Fabry gene therapy program on FDA clinical hold
Pharmaceutical Technology
FEBRUARY 10, 2023
4D Molecular Therapeutics (4DMT), the California-based biotechnology company focused on developing gene therapies for rare and large market diseases, has had the FDA place a clinical hold onto its Fabry disease (FD) gene therapy program (4D-310).
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Sartorius upscales gene therapy prowess with $2.6bn Polyplus acquisition
Pharmaceutical Technology
APRIL 3, 2023
The deal will see Polyplus join the German life science group’s portfolio allowing the latter to leverage expertise in transfection reagents and plasmid DNA for gene therapy. Polyplus, based in Strasbourg, France, produces key components in the production of viral vectors used in cell and gene therapies.
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Amplo Biotechnology receives NIH-NIAMS grant for gene therapy AMP-201
Pharmaceutical Technology
MAY 30, 2023
Amplo Biotechnology has received a fast track phase I/II STTR grant from the NIH-NIAMS [National Institutes of Health’s National Institute of Arthritis and Musculoskeletal and Skin Diseases] for its gene therapy AMP-201. The company will receive substantial funding to advance AAV-ColQ gene therapy.
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Lilly agrees to acquire gene therapy developer Akouos for $610m
Pharmaceutical Technology
OCTOBER 19, 2022
Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss. It has combined expertise in otology, gene therapy and inner ear drug delivery to meet the requirements of individuals with disabling hearing loss globally.
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Forge Biologics joins BGTC for new AAV gene therapies development
Pharmaceutical Technology
MAY 24, 2023
Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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5 ways experience matters in gene therapy manufacturing
Bio Pharma Dive
AUGUST 21, 2023
By partnering with an accomplished CTDMO, gene therapy innovators can benefit from guidance and support.
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Generation bio cuts 40% of staff in blow for non-viral gene therapy sector
Pharmaceutical Technology
NOVEMBER 30, 2023
The move is a strategic response amid challenges faced by the non-viral gene therapy sector, with several startups closing this year.
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Pfizer’s hemophilia B gene therapy inches closer to regulatory approval
Bio Pharma Dive
JUNE 27, 2023
The treatment could become Pfizer’s first marketed gene therapy, an area the pharma has poured significant resources into in recent years.
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Astellas takes stake in gene therapy developer Taysha
Bio Pharma Dive
OCTOBER 24, 2022
Already an investor in gene therapy, the Japanese pharma will buy 15% of the Texas biotech’s stock and gain options to two gene therapies for central nervous system disorders.
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Astellas to support development of Taysha’s gene therapy programmes
Pharmaceutical Technology
OCTOBER 25, 2022
Ast ellas Pharma has announced plans to make a strategic investment to back the development of Taysha Gene Therapies’ adeno-associated virus (AAV) development programmes for Rett syndrome and giant axonal neuropathy (GAN). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Cell and gene therapy is coming of age
Bio Pharma Dive
JULY 24, 2023
Outsourcing in the cell and gene therapy industry is booming and no longer considered just a “trend.”
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Addressing manufacturing challenges in gene therapy development
Bio Pharma Dive
MARCH 20, 2023
As new gene therapies are developed to treat a growing number of indications, patients may soon have access to novel treatment options and potential cures.
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AstraZeneca buys Pfizer’s early gene therapy work for up to $1B
Bio Pharma Dive
JULY 28, 2023
The British pharma is deepening its investment in genetic medicine through a deal for a group of Pfizer’s gene therapy delivery tools, while the New York giant cuts back.
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Freeline cuts more jobs and halts Fabry gene therapy work
Bio Pharma Dive
APRIL 4, 2023
biotech will lay off 30% of its workforce and focus on an experimental Gaucher disease treatment, five months after an initial restructuring that cut a hemophilia gene therapy.
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Royalty Pharma buys into Ferring’s cancer treatment in first gene therapy deal
Bio Pharma Dive
AUGUST 24, 2023
The deal, worth up to $500 million, is a bet by the drug rights dealmaker on the uncertain commercial prospects of Ferring's gene therapy Adstiladrin.
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Prevail, Capsida partner to develop gene therapies for nervous system diseases
Bio Pharma Dive
JANUARY 4, 2023
The Lilly-owned subsidiary will pay $55 million to gain access to Capsida’s AAV gene therapy technology in a deal aimed at central nervous system disorders.
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Alexion to acquire Pfizer’s rare disease gene therapies for $1bn
Pharmaceutical Technology
JULY 31, 2023
Alexion has signed a definitive agreement for acquiring and licencing Pfizer’s early-stage gene therapy portfolio for rare diseases.
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5 questions facing gene therapy in 2022
Bio Pharma Dive
JANUARY 13, 2022
As record levels of money pour into gene therapy research, biotechs are under increased pressure to address issues related to safety, delivery and overlapping pipelines.
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Krystal Biotech receives FDA approval for DEB topical gene therapy
Pharmaceutical Technology
MAY 26, 2023
Krystal Biotech has received approval from the US Food and Drug Administration for topical gene therapy VYJUVEK to treat dystrophic epidermolysis bullosa (DEB) in adults and in children aged six months and above. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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FDA suggests ways to curb constraints with rare disease gene therapy trials
Pharmaceutical Technology
FEBRUARY 8, 2023
On February 7, at a town hall organised to discuss clinical trial designs for gene therapies, FDA experts pushed pharma players to look for ways to establish clinical effectiveness despite the challenges in recruiting patients with rare diseases.
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Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy
Bio Pharma Dive
MAY 17, 2023
Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.”
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Ginkgo grows its gene therapy offerings with StrideBio deal
Bio Pharma Dive
APRIL 5, 2023
The deal hands Ginkgo technology for discovering and engineering capsids — the outer shell that protects the helpful genetic material in gene therapies.
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uniQure signs license deal for Apic Bio’s gene therapy for SOD1-ALS
Pharmaceutical Technology
JANUARY 31, 2023
Gene therapy company uniQure has entered into a global licensing agreement with Apic Bio for APB-102 to treat patients with amyotrophic lateral sclerosis (ALS) caused by mutations in superoxide dismutase 1 (SOD1). APB-102 is designed to be a one-time, intrathecally administered gene therapy for ALS patients.
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FDA grants approval for CSL’s haemophilia B gene therapy
Pharmaceutical Technology
NOVEMBER 23, 2022
The US Food and Drug Administration (FDA) has granted approval for CSL Behring’s adeno-associated virus vector-based gene therapy, Hemgenix (etranacogene dezaparvovec), to treat haemophilia B (congenital Factor IX deficiency) in adult patients. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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Research consortium picks eight rare diseases to advance gene therapies
Pharmaceutical Technology
MAY 16, 2023
The Foundation for the National Institutes of Health (FNIH) has announced its plans to prioritise eight rare diseases to provide industry standards for manufacturing, preclinical testing and product analytical testing for gene therapy development. This will include pairing up indications with manufacturers amongst the BGTC’s partners.
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New facility boosts UK’s cell and gene therapy manufacturing capacity
Pharmaceutical Technology
MARCH 10, 2023
On 10 March, the National Health Service Blood and Transplant (NHSBT) opened a new Clinical Biotechnology Centre (CBC) with the aim of improving the UK’s ability to develop and manufacture cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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