pharmaphorum

FEBRUARY 27, 2024

Looking to the future of clinical trials: Gene therapy, precision medicine, and the ongoing quest for rare disease solutions Mike.Hammerton Tue, 27/02/2024 - 12:30 Bookmark this

Gene Therapy 122

Gene Therapy Clinical Trials Clinical Trials Gene 122

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

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.

Gene Editing 64

Gene Editing Gene Medicine Genetics 64

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 258

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.

Gene Therapy 278

Gene Therapy Gene Development Genetics 278

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.

Gene Therapy 256

Gene Therapy Gene Development Manufacturing 256

Bio Pharma Dive

JUNE 26, 2021

Results published in The New England Journal of Medicine offer the first clinical evidence that CRISPR gene editing inside the body can be safe and effective, a culmination of years of scientific research.

Gene Editing 363

Gene Editing Gene Medicine Research 363

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

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.

Gene Editing 278

Gene Editing Gene Medicine 278

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

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

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

Gene Therapy Gene Development Manufacturing 242

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

Gene Therapy Gene Gene Editing Manufacturing 243

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.

Genome 130

Genome Genomics Medicine Development 130

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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Gene Therapy Gene Research Clinical Trials 227

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.

Gene Therapy 264

Gene Therapy Manufacturing Gene Clinical Trials 264

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.

Gene Therapy 293

Gene Therapy Gene Genetics Medicine 293

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.

Gene Editing 356

Gene Editing Medicine Gene 356

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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Gene Therapy Gene Genetics Medicine 200

Bio Pharma Dive

APRIL 1, 2024

The FDA could soon expand use of two multiple myeloma cell therapies as well as a top-selling medicine from Sanofi and Regeneron, while Pfizer’s years of gene therapy work might finally pay dividends.

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Gene Therapy Gene Medicine 300

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

Medicine 105

Medicine Gene Editing Genetics Gene 105

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

Gene Therapy 300

Gene Therapy Gene Genetics Medicine 300

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.

Gene Therapy 246

Gene Therapy Gene Genome Genomics 246

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.

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Gene Editing Medicine Gene Development 306

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

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 334

Pharmaceutical Technology

NOVEMBER 9, 2022

On November 2, the Institute for Clinical and Economic Review (ICER) released its updated evidence aimed at measuring the clinical effectiveness and cost of the two haemophilia gene therapies. An approval decision on the gene therapy, also known as EtranaDez, is expected by the end of this month. million price tag.

Gene Therapy 278

Gene Therapy Gene FDA Approval Medicine 278

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.

Gene Therapy 294

Gene Therapy Gene Medicine 294

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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Gene Editing Gene Gene Therapy In-Vivo 165

Fierce Pharma

MARCH 14, 2024

After charting multiple personalized medicine expansions in recent years, contract manufacturer Charles River Laboratories is back with another gene therapy pact—this time centered on an adeno-asso | After charting multiple personalized medicine expansions in recent years, contract manufacturer Charles River Laboratories is back with another gene therapy (..)

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Gene Therapy Contract Manufacturing Gene Production 95

BioPharma Reporter

MARCH 7, 2024

In the realm of modern medicine, few advancements have inspired and challenged the industry quite like gene therapy.

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Gene Therapy Gene Medicine 116

Pharmaceutical Technology

FEBRUARY 27, 2024

Making therapies feasible from biological, regulatory, and commercial standpoints drives scalable genetic medicine.

Gene Therapy 246

Gene Therapy Gene Genetics Medicine 246

BioSpace

JUNE 12, 2022

Shape Therapeutics is evolving programmable RNA medicines that push the limits of current gene editing capabilities. BioSpace spoke with CSO David Huss.

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Gene Editing RNA Gene Medicine 98

Bio Pharma Dive

FEBRUARY 8, 2024

The company is one of three preclinical biotechs to successfully go public since last summer, but it pulled in significantly less than others who’ve debuted with more advanced medicines.

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Gene Editing Gene Medicine 200

Pharma Times

JANUARY 28, 2022

Gene luminaries Oxford Biomedica reach agreement with Homology Medicines to develop super group

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Gene Medicine Development 103

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.

Gene Editing 306

Gene Editing Medicine Gene Research 306

Fierce Pharma

DECEMBER 8, 2023

| Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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Gene Therapy FDA Approval Gene Gene Editing 129