Pharmaceutical Technology

OCTOBER 22, 2024

Editas is partnering its CRISPR platform with Genevant’s LNP technology to develop novel gene editing medicines in a deal worth $283m.

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Gene Editing Gene Development Medicine 244

Bio Pharma Dive

OCTOBER 20, 2022

The gene editing startup is one of less than two dozen biotech companies to debut on Wall Street this year, as the pace of IPOs remains far off last year’s trajectory.

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Gene Editing Medicine Gene 355

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 Akouos focuses on developing adeno-associated viral gene therapies to treat inner ear conditions, including sensorineural hearing loss.

Gene Therapy 298

Gene Therapy Gene Development Genetics 298

Pharmaceutical Technology

JANUARY 16, 2023

CARsgen Therapeutics has announced a col labor ation with Huadong Medicine to commercialise zevorcabtagene autoleucel (zevor-cel), CT053, in mainland China. Under the collaboration, Huadong Medicine will have the exclusive right to commercialise CARsgen’s CT053 in mainland China. By Cytiva Thematic.

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Medicine Gene Therapy Production Gene 298

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 328

Genetics Medicine Development Gene Therapy 328

Bio Pharma Dive

JULY 12, 2022

A patient in New Zealand became the first person to receive the biotech’s medicine, which uses base editing to turn off a specific gene in the liver and thereby lower cholesterol.

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Gene Editing Gene Medicine 273

Bio Pharma Dive

APRIL 24, 2023

Submission of the application comes weeks after rivals Vertex and CRISPR filed their gene editing medicine for the disease with the agency.

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Gene Editing Gene Gene Therapy FDA Approval 256

Pharmaceutical Technology

APRIL 19, 2024

Paolo Morgese, vice president of European public affairs at the Alliance for Regenerative Medicine, discusses the barriers affecting access to gene therapies.

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Gene Therapy Gene Medicine 237

Bio Pharma Dive

JULY 14, 2021

Formed around research from the Broad Institute's David Liu and Andrew Anzalone, Prime has attracted significant funding from a wide range of high-profile biotech backers.

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Gene Editing Medicine Gene Research 300

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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Gene Therapy Genetics Medicine Gene 304

Pharmaceutical Technology

AUGUST 25, 2022

The European Commission (EC) has granted conditional marketing authorisation (CMA) for BioMarin Pharmaceutical ’s gene therapy, Roctavian (valoctocogene roxaparvovec), to treat adults with severe haemophilia A (congenital Factor VIII deficiency). Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Clinical Trials Clinical Trials 264

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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Gene Therapy Gene Genetics Medicine 246

Bio Pharma Dive

AUGUST 17, 2022

The regulator cleared the biotech’s medicine Zynteglo for transfusion-dependent beta thalassemia, giving patients a powerful new treatment option. But it will come at a very high cost of $2.8 million in the U.S.

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Gene Therapy Gene Regulation Medicine 363

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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Gene Therapy Gene Clinical Trials Clinical Trials 264

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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Gene Therapy Gene Development Manufacturing 256

Bio Pharma Dive

NOVEMBER 17, 2022

While the biotech will not develop the gene editing therapy further on its own, CEO Gilmore O’Neill claimed the data are still a meaningful demonstration of what CRISPR can accomplish.

Gene Editing 299

Gene Editing Medicine Gene Development 299

Bio Pharma Dive

MARCH 24, 2021

A group of drug companies, including Novartis, Takeda and Roche's Genentech, have agreed to collaborate with Vineti to usher in new identification standards for complex medicines.

Gene Therapy 357

Gene Therapy Gene Medicine Drugs 357

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.

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Gene Editing Gene Genetics Medicine 294

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 30, 2024

Researchers see a greater need for new generation therapeutics to propel precision medicine. The development of innovative therapeutic approaches in healthcare encompass a variety of fields, including gene therapy, personalized medicine, immunotherapy, and advanced biologics.

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Medicine Research Gene Therapy Genetics 189

Bio Pharma Dive

JUNE 29, 2023

After a prolonged journey, the medicine, known as Roctavian, is now cleared for certain patients with hemophilia A, the more common form of the rare bleeding disorder.

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Gene Therapy FDA Approval Gene Medicine 330

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. Personalised medicines will also be developed at the centre.

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Gene Therapy Manufacturing Gene Clinical Trials 264

Bio Pharma Dive

JANUARY 7, 2025

The three-year partnership is another example of Vertex’s interest in improving on Casgevy, its CRISPR medicine for sickle cell and beta thalassemia.

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Gene Editing Gene Research Medicine 288

Bio Pharma Dive

APRIL 11, 2021

Study results presented at AACR showed Retevmo shrank tumors in nine different cancers, building a case for the type of tissue-agnostic label that only three other medicines have secured.

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Gene Medicine Drugs 323

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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Gene Therapy Gene Genetics Medicine 341

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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Gene Therapy Gene Trials Clinical Trials 264

Bio Pharma Dive

DECEMBER 9, 2021

The amount of blood clotting protein found in patients who received the medicine was nearly the same at six months and 18 months post-treatment, according to results disclosed Thursday.

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Gene Therapy Gene Protein Medicine 327

Bio Pharma Dive

SEPTEMBER 26, 2022

The startup, which launched last year with $315 million in funding and plans to advance a new form of gene editing, is one of the first big venture-backed biotechs in several months to seek an IPO.

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Gene Editing Medicine Gene 282

Pharmaceutical Technology

MARCH 10, 2023

Cell One Partners has announced a strategic collaboration with the Center for Breakthrough Medicines (CBM) to expedite the development and commercialisation of cell and gene therapies. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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Gene Therapy Gene Manufacturing Development 246

Bio Pharma Dive

OCTOBER 27, 2022

Landmark Bio, armed with $75 million and a wide range of backers, is the latest company to emerge with plans to open a “bottleneck” slowing development of the complex medicines.

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Gene Medicine Development 327

Bio Pharma Dive

JUNE 22, 2023

The cost makes Sarepta’s treatment, called Elevidys, among the most expensive medicines in the world. But company executives said they don’t expect significant insurer pushback.

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Gene Therapy Gene Medicine 287

AuroBlog - Aurous Healthcare Clinical Trials blog

SEPTEMBER 17, 2024

Biology is opening up new frontiers in medicine. We are witnessing a revolution in healthcare, driven by advances in genetics, Omics, RNA and CRISPR gene-editing technology, to deliver precision and personalised medicine, said Kiran Mazumdar-Shaw, executive chairperson, Biocon and Biocon Biologics.

Genetic Disease 189

Genetic Disease Genetics Gene Editing Medicine 189

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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Gene Therapy Gene Gene Editing Development 243

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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Gene Therapy Gene Genome Genomics 246

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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Gene Therapy Gene Development Manufacturing 242

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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Gene Therapy Gene Protein Sales 246

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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Gene Editing Gene Genetics Medicine 310

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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Gene Therapy Gene Development Engineer 237