Regeneron expands in gene editing with Mammoth deal
Bio Pharma Dive
APRIL 25, 2024
“With each passing year, we're more committed to becoming a serious player in the genetic medicine space,” a Regeneron executive said.
Lilly and ProQR to expand genetic medicine development agreement
Pharmaceutical Technology
DECEMBER 23, 2022
Eli Lilly and Company has expanded a licencing and partnership agreement with ProQR Therapeutics to discover, develop and market new genetic medicines. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. The companies entered the initial agreement in September last year. By Cytiva Thematic.
Genetics 
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Moderna and Generation Bio partner for genetic medicines development
Pharmaceutical Technology
MARCH 24, 2023
Moderna has entered a strategic partnership with Generation Bio for the development of non-viral genetic medicines. Moderna’s biological and technical expertise will be combined with core technologies of the non-viral genetic medicine platform from Generation Bio.
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Cell and gene therapies: why advanced medicines call for specialised logistics
Pharmaceutical Technology
JULY 7, 2022
Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. A new frontier in cancer research.
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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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Lilly gene therapy finding puts focus on hearing loss treatment pipeline
Bio Pharma Dive
JANUARY 24, 2024
An 11-year-old boy born deaf can hear after receiving Lilly's genetic medicine. Others, including biotechs in China and Regeneron in the U.S., are taking aim at the same drug target.
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Yet another gene therapy developer turns to layoffs
Bio Pharma Dive
APRIL 1, 2022
Taysha Gene Therapies is narrowing its research efforts and cutting around 35% of its workforce, the latest in a series of layoffs announced by makers of genetic medicines.
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Pricey new gene therapies for sickle cell pose access test
Bio Pharma Dive
DECEMBER 8, 2023
million, while a competing genetic medicine also cleared Friday is priced at $3.1 Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2
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Gene-editing firm Prime Medicine files $140m public offer
pharmaphorum
FEBRUARY 15, 2024
Gene-editing biotech Prime Medicine has priced its IPO, hoping to raise $140m to advance its pipeline of one-shot therapies for severe genetic disorders.
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Operational risks with gene therapies should improve with time, exec says
Pharmaceutical Technology
FEBRUARY 27, 2024
Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.
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Orchard sets out to sell world’s priciest gene therapy
Bio Pharma Dive
MARCH 20, 2024
million list price, the highest of any genetic medicine to come to market. Orchard is counting on the long-term data it’s accrued to convince insurers to cover Lenmeldy’s $4.25
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Lilly agrees to acquire gene therapy developer Akouos for $610m
Pharmaceutical Technology
OCTOBER 19, 2022
Eli Lilly and Company has signed a definitive agreement for the acquisition of all outstanding shares of precision genetic medicine firm Akouos for a total deal value of up to nearly $610m or up to $15.50 It is a gene therapy intended to treat hearing loss due to otoferlin gene (OTOF) mutations. for each share in cash.
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Regeneron, Intellia target neurological diseases in expanded gene editing deal
Bio Pharma Dive
OCTOBER 3, 2023
The longtime partners believe that, by combining their technologies, they can create “in vivo” genetic medicines for nervous system and muscular disorders.
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Lilly pays $880M for Prevail, pushing further into genetic medicine
Bio Pharma Dive
DECEMBER 15, 2020
The acquisition marks a significant bet by Lilly on gene therapy's potential to address tough-to-treat neurological disorders like Parkinson's disease.
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Verve partners with Lilly on gene editing therapy for heart disease
Bio Pharma Dive
JUNE 15, 2023
Lilly will pay the genetic medicine specialist $60 million upfront to work together on a treatment targeting a risk factor known as Lp(a).
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CRISPR Pioneer Goes Small to Transform Genetic Medicine with Epigenetic Engineering
BioSpace
JULY 11, 2022
Epic Bio aims to transform genetic medicine by developing a new class of drugs that target the epigenome to alter gene expression.
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Ginkgo adds gene editing tools in buyout of Feng Zhang-founded startup
Bio Pharma Dive
FEBRUARY 28, 2024
The acquisition of Proof Diagnostics, which was formed to develop a COVID-19 test, gives Ginkgo a library of novel enzymes that can be used to make genetic medicines.
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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. Topic sponsors are not involved in the creation of editorial content.
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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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Prime Medicine joins the biotech IPO queue, seeking $140m
pharmaphorum
FEBRUARY 15, 2024
Gene-editing biotech Prime Medicine has priced an initial public offering, hoping to raise $140 million to advance its pipeline of one-shot therapies for severe genetic disorders. The Cambridge, Massachusetts-based company is offering 19.2 million shares at $6.25
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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. In June 2023, the U.S.
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Hopewell Therapeutics raises funds for genomic medicines development
Pharmaceutical Technology
JUNE 8, 2023
Biotechnology company Hopewell Therapeutics has raised $25m in seed financing to accelerate the development of next-generation lipid nanoparticles for targeted delivery of genomic medicines. Hopewell Therapeutics is engaged in discovering, synthesising and developing advanced ttLNPs to provide next-generation genomic medicines.
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Start-up aims to advance transformative genetic medicines
Drug Discovery World
FEBRUARY 16, 2023
Challenges remain with current delivery approaches, and genetic medicines applications have been generally restricted to the liver and ex vivo settings. Aera’s technology also includes a licensed therapeutic enzyme platform based on the discovery of novel, compact, and programmable gene editing enzymes.
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Regeneron, in search of an eye gene therapy, turns to a young biotech
Bio Pharma Dive
APRIL 6, 2022
Facing mounting competition to its top-selling drug Eylea, Regeneron is teaming up with ViGeneron to develop a genetic medicine for an inherited retinal disease.
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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.
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Ovid turns to gene therapy startup to restock drug pipeline
Bio Pharma Dive
AUGUST 24, 2022
The New York biotech will invest in and develop up to three drugs with Gensaic, an emerging startup aiming to use the viruses that infect bacteria to deliver genetic medicines.
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AGTC yields to biotech downturn with gene therapy buyout deal
Bio Pharma Dive
OCTOBER 24, 2022
The biotech is the latest in a growing list of genetic medicine developers to sell themselves near record stock lows amid a challenging funding environment.
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Innovation in immuno-oncology: Leading companies in gene therapy delivery using viral vectors
Pharmaceutical Technology
FEBRUARY 15, 2023
In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Immuno-oncology in Pharmaceuticals: Gene therapy delivery using viral vectors. Sanofi is one of the leading patent filers working with viral vectors.
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New company will focus on genetic medicines for neural circuit dysfunction
Drug Discovery World
MAY 19, 2023
Sania Therapeutics, a new company focused on developing genetic medicines for neural circuit dysfunction, has launched at the American Society of Gene & Cell Therapy (ASGCT) conference. A second platform, Gre-Scan, is designed for the high throughput screening of gene regulatory elements.
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Capsida Biotherapeutics and Prevail to develop CNS gene therapies
Pharmaceutical Technology
JANUARY 5, 2023
Capsida Biotherapeutics and Eli Lilly and Company ’s wholly owned subsidiary Prevail Therapeutics have announced a partnership for the development of non-invasive gene therapies for central nervous system (CNS) diseases. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva. By Cytiva Thematic.
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Sania Therapeutics launches to advance novel medicines
BioPharma Reporter
MAY 18, 2023
Sania Therapeutics, a new biotech company focused on developing genetic medicines for neural circuit dysfunction, has launched by unveiling its suite of proprietary patented platforms at the American Society of Gene & Cell Therapy (ASGCT) conference.
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Italfarmaco’s Duvyzat Wins FDA Approval as First Nonsteroidal Treatment for All Genetic Variants of DMD
XTalks
MARCH 28, 2024
Duvyzat, a histone deacetylase (HDAC) inhibitor that works to reduce inflammation and muscle loss, is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD. Progressive muscle weakness in the disease is caused by genetic mutations in the dystrophin gene that lead to a lack of functional dystrophin protein.
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Lenmeldy, First Approved MLD Gene Therapy for Children, Becomes Most Expensive Drug in the US at $4.25 Million
XTalks
MARCH 21, 2024
The US Food and Drug Administration (FDA) has awarded approval to Orchard Therapeutics for its gene therapy Lenmeldy (atidarsagene autotemcel) for the treatment of children with pre-symptomatic late infantile, pre-symptomatic early juvenile or early symptomatic early juvenile metachromatic leukodystrophy (MLD).
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Genetically engineered dendritic cells enhance lung cancer therapy
Drug Discovery World
APRIL 9, 2024
To help enhance the effectiveness of immune checkpoint blockade, the UCLA team explored injecting immune-stimulating, chemokine gene-engineered dendritic cells directly into the tumour. The scientists genetically modified dendritic cells to produce CXCL9 and CXCL10 and then injected these directly into the tumours in mouse models of NSCLC.
Pfizer opens gene therapy plant in $800M North Carolina expansion
Bio Pharma Dive
DECEMBER 16, 2021
The new facility cost nearly $70 million to build, and is part of a major push by the pharma giant to become a leader in genetic medicine manufacturing.
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BUSM Researchers Gain Insight into Key Genetic Driver of Alzheimer’s
BioSpace
JUNE 23, 2022
The Boston University School of Medicine has unraveled details about a known genetic component for the development of Alzheimer’s disease (AD): the APOE4 gene.
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CRISPR, with new partner, to develop gene editing therapies for ALS, nerve disorder
Bio Pharma Dive
JUNE 15, 2021
A deal with biotech startup Capsida, which recently raised $140 million, gives CRISPR access to a Caltech technology that aims to improve the delivery of genetic medicines.
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Beqvez Becomes Pfizer’s First Approved Gene Therapy After Nod from Health Canada
XTalks
JANUARY 4, 2024
Pfizer has kickstarted the new year with its first-ever gene therapy approval, awarded by Health Canada to the company’s Beqvez (fidanacogene elaparvovec) for the treatment of hemophilia B. There is a significant focus on developing gene therapies as longer-term solutions for the disease.
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Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation
Pharmaceutical Technology
MAY 24, 2023
It can correct several disease elements associated with PKD, a rare, monogenic red blood cell disorder caused by a mutation in the PKLR gene. The RMAT designation programme is intended to accelerate the drug’s development and review processes for products, including gene therapies.
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A gene therapy pipeline takes shape for a cluster of rare diseases
Bio Pharma Dive
FEBRUARY 12, 2021
Lysosomal storage disorders, diseases of cellular metabolism gone wrong, are the focus of a fast-expanding lineup of experimental genetic medicines, a research boom that recalls biotech's roots.
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Head of FDA gene therapy office set to retire in March
Bio Pharma Dive
FEBRUARY 1, 2023
Wilson Bryan, a key decision maker in the agency’s oversight of genetic medicines, is expected to leave next month amid an organizational revamp of his office, the FDA confirmed.
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How Pharmacogenomics Can Benefit Your Clinical Trial
Worldwide Clinical Trials
MARCH 20, 2024
In the increasingly advancing world of medicine, personalized care is a key factor that can progress your drug development efforts. As the study of how an individual’s genes affect their response to drugs, pharmacogenomics is a critical factor in developing effective and safe drugs and dosages in clinical trials.
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Great Britain first to approve CRISPR-based gene therapy
Drug Discovery World
NOVEMBER 16, 2023
The Medicines and Healthcare products Regulatory Agency (MHRA) has authorised the world’s first gene therapy for sickle-cell disease (SCD) and transfusion-dependent β-thalassemia (TDT). Casgevy is designed to work by editing the faulty gene in a patient’s bone marrow stem cells so that the body produces functioning haemoglobin.
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