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.
Gene Related Topics 
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.
Gene Therapy 
281
281 Join 32,000+
Insiders
Sign Up for our Newsletter
This site is protected by reCAPTCHA and the Google Privacy Policy and Terms of Service apply.
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
Gene Therapy 241
241 
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.
Gene Therapy 264
264 
Making the Case for Case Management for Cell and Gene Therapies
Advertisement
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.
The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment
Worldwide Clinical Trials
NOVEMBER 27, 2023
Casgevy, the commercial product formerly known as exa-cel, is administered by taking stem cells out of a patient’s bone marrow and editing a gene in the cells in a laboratory, with the modified cells then infused back into the patient after conditioning treatment to prepare the bone marrow.
Gene Editing 189
189 
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.
Gene Therapy 271
271 
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.
Gene Therapy 246
246 
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.
Gene Therapy 246
246 
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.
Gene Therapy 256
256 
3 Key Takeaways from Global Genes Summit 2023
Worldwide Clinical Trials
OCTOBER 24, 2023
Global Genes is a rare disease umbrella organization dedicated to eliminating the burdens and challenges of rare diseases for patients and families globally. Over the years, I have attended several Global Genes Summits. The post 3 Key Takeaways from Global Genes Summit 2023 appeared first on Worldwide Clinical Trials.
Gene 130
130 
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.
Gene Therapy 305
305 
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. In 2022, Avrobio’s investigational gene therapy, AVR-RD-04, received a rare paediatric disease designation from the US Food and Drug Administration.
Gene Therapy 258
258 
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.
Gene Therapy 246
246 
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.
Gene Therapy 330
330 
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.
Gene Therapy 246
246 
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.
Gene Therapy 246
246 
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.
Gene Therapy 246
246 
Using twin viruses, startup AAVantgarde aims to extend gene therapy’s reach
Bio Pharma Dive
JUNE 6, 2023
The biotech has raised about $65 million to test two ways to deliver larger genes into the body, each of which could help gene therapy treat more diseases.
Gene 260
260 
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.
Gene Therapy 271
271 
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.
Gene Therapy 246
246 
Cure Genetics and Frametact seek gene cures for neurological ailments
Pharmaceutical Technology
SEPTEMBER 15, 2023
Cure Genetics has entered a partnership and licensing deal with Frametact to develop gene therapy for familial neurological ailments.
Genetics 246
246 
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.
Gene Therapy 298
298 
ElevateBio raises funds to advance cell and gene therapies
Pharmaceutical Technology
MAY 25, 2023
ElevateBio has raised $401m in a Series D financing round for advancing its technology platforms to expedite the design, production and development of cell and gene therapies. The technology platforms include the Life Edit gene editing platform, an RNA, cell, protein, vector engineering and induced pluripotent stem cells (iPSCs) platform.
Gene Therapy 243
243 
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).
Gene Therapy 264
264 
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.
Gene Therapy 246
246 
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.
Gene Therapy 244
244 
Moderna inks another gene editing deal
Bio Pharma Dive
FEBRUARY 22, 2023
The messenger RNA specialist said Wednesday it is teaming up with Life Edit Therapeutics to develop therapies that can modify genes “in vivo.”
Gene Editing 259
259 
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. DEB, a painful skin condition, is caused due to mutations in the COL7A1 gene resulting in the lack of functional type VII collagen.
Gene Therapy 264
264 
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.
Gene Therapy 242
242 
FDA won’t hold advisory meeting for Bluebird’s sickle cell gene therapy
Bio Pharma Dive
AUGUST 16, 2023
The decision stands in contrast to the agency’s plans for a would-be rival gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that is also under a regulatory review.
Gene 245
245 
Sanofi expands research deal with gene editing startup Scribe
Bio Pharma Dive
JULY 17, 2023
Scribe will receive $40 million upfront from Sanofi in a collaboration initially focused on developing an in vivo gene editing treatment for sickle cell disease.
Gene Editing 237
237 
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.
Gene Therapy 273
273 
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.
Gene Therapy 264
264 
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.
Gene Therapy 130
130 
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.
Gene Therapy 246
246 
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.
Gene Therapy 271
271 
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.
Gene Therapy 246
246 
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.
Gene Therapy 277
277 
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.
Gene Therapy 286
286 
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.
Gene Therapy 227
227 
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.
Gene Therapy 264
264 
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.”
Gene Therapy 246
246 
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.
Gene Therapy 352
352 
Let's personalize your content