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. ElevateBio intends to use the funds to advance its genetic medicine current good manufacturing practice (cGMP) and process development business, BaseCamp.

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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.

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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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Bio Pharma Dive

DECEMBER 8, 2023

Casgevy, the first CRISPR therapy approved by the FDA, will cost $2.2 million, while a competing genetic medicine also cleared Friday is priced at $3.1

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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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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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Pharmaceutical Technology

FEBRUARY 27, 2024

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

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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.

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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 therapy has also received orphan drug and fast-track designations. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.

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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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Drug Discovery World

MAY 8, 2024

Recombinant adeno-associated viruses (rAAVs) are considered a promising delivery system for gene therapy medicines. The post How can long-read nanopore sequencing support gene therapy delivery? appeared first on Drug Discovery World (DDW).

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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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Pharmaceutical Technology

JUNE 19, 2023

Although only a small number of gene therapies have reached the market thus far, the industry is poised to grow quickly over the next few years. According to GlobalData’s clinical trials database, there are currently 1,231 planned and ongoing trials for gene therapies and gene-modified cell therapies alone.

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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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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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Drug Discovery World

JUNE 23, 2023

Sarepta Therapeutics’ Elevidys has become the first gene therapy for Duchenne muscular dystrophy (DMD) to gain marketing authorisation in the US. It is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

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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). It is estimated that this includes around 2,000 patients in the UK.

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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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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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XTalks

MAY 17, 2023

The Foundation for the National Institutes of Health (FNIH) announced this week that the Accelerating Medicines Partnership Bespoke Gene Therapy Consortium (AMP BGTC) has selected eight rare diseases for its clinical trial portfolio.

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XTalks

MAY 26, 2023

Krystal Biotech’s Vyjuvek has been awarded US Food and Drug Administration (FDA) approval to make it the first topical gene therapy for the treatment of wounds in patients with the rare, often debilitating skin disease dystrophic epidermolysis bullosa (DEB). As a topical treatment, it is also the first readily redosable gene therapy.

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Drug Discovery World

AUGUST 7, 2023

Evox Therapeutics has agreed a research collaboration and option agreement with the Icahn School of Medicine at Mount Sinai (Icahn Mount Sinai) in New York, NY, US. The collaboration will work on developing exosome-encapsulated AAV (exoAAV) vectors as a novel gene delivery technology aimed at improving treatments for heart disease.

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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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Drug Discovery World

OCTOBER 18, 2023

Sara Donnelly, Director of Research Planning and Business Development at PhoenixBio USA explores why the right pre-clinical model is essential for teams wanting to advance adeno-associated virus vector-based gene therapies.

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Drug Discovery World

FEBRUARY 1, 2024

Vivet is a clinical stage biotech company developing gene therapies for rare inherited metabolic disorders. CTX is a rare autosomal recessive genetic disorder that affects the body’s ability to metabolise fats known as cholesterols. Vivet Therapeutics has received €4.9

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XTalks

APRIL 24, 2023

Approximately 72 percent of rare diseases are genetic, and around 70 percent of rare genetic diseases emerge in childhood. Out of over 7,000 rare diseases, only 5 percent (or less) of rare diseases are thought to have approved treatment options, known as “orphan” therapies.

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BioPharma Reporter

MAY 6, 2022

CDMO Matica Bio has opened a new 45,000-square-foot facility dedicated to the production of viral vectors and cell-based products used in cell and gene therapies, vaccines, oncolytic therapies and other genetic medicines.

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Camargo

MAY 2, 2021

Advances in scientific knowledge and growth in the cell and gene therapy space have led to a new and exciting era of medicine for patients, as well as a new motivation for regulators to provide clear, efficient pathways for product developers. Background: The Advancement of Cell and Gene Therapies.

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Drug Discovery World

AUGUST 23, 2022

A Phase I/II clinical trial targeting a treatment for Huntington’s Disease (HD) using a gene therapy has been given the clearance to go ahead in France. . The disease is caused by anomalous repeating mutations in the huntingtin gene leading to abnormal protein aggregates in nerve cells.

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XTalks

JUNE 30, 2023

It’s been a big week for cell and gene therapy approvals in the US, including a much-awaited approval for one to treat hemophilia A, the most common form of hemophilia. Hemophilia A is a rare genetic bleeding disorder that is caused by a mutation in the gene that encodes the key blood clotting protein factor VIII (FVIII).

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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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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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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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pharmaphorum

FEBRUARY 23, 2022

Takeda has forged another alliance as it continues a push into gene therapy, agreeing a deal worth up to $2 billion with Code Biotherapeutics for opt-in rights to four candidates for rare diseases. The post Takeda grows in gene therapies again with $2bn Code Bio deal appeared first on. Last October it signed a $3.6

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Medical Xpress

DECEMBER 21, 2022

Ten young children born without functioning immune systems and lacking the ability to fight infections are on track for healthier lives thanks to a new gene therapy treatment pioneered at UC San Francisco, reports a Dec. 22 study in the New England Journal of Medicine.

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Bio Pharma Dive

MARCH 15, 2021

Softbank and Fidelity joined a large group of investors in a $525 million Series C investment into Elevate, a high-powered genetic medicine startup with an unusual business model.

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Drug Discovery World

DECEMBER 11, 2023

The US Food and Drug Administration (FDA) has approved the first cell-based gene therapies for the treatment of sickle cell disease (SCD), Casgevy and Lyfgenia. Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is also the first FDA-approved treatment to use CRISPR gene editing technology.

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