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.

FDA Approval 105

FDA Approval Genetics Gene Therapy Gene 105

BioSpace

SEPTEMBER 15, 2020

Clinical-stage biopharmaceutical firm ESCAPE Bio has snagged a $73 million crossover financing deal, led by Wellington Management Company, which will help advance the company’s two clinical development programs of precision neurology medicines for patients with genetic neurodegeneration.

Clinical Development 97

Clinical Development Development Genetics Medicine 97

Pharmaceutical Technology

JUNE 6, 2023

Alkeus Pharmaceuticals has raised $150m in Series B financing to support the registration and launch of gildeuretinol (ALK-001) to treat Stargardt disease, a genetic cause of blindness in children and young adults. The genetic cause is well characterised, but to date there is no effective treatment.

Genetics 279

Genetics Life Science Trials Clinical Development 279

Medical Xpress

APRIL 26, 2023

A successful gene therapy trialed at Michigan State University in dogs with an inherited eye disease is ready to be developed for clinical use in human patients with a rare condition called retinitis pigmentosa.

Clinical Development 133

Clinical Development Gene Therapy Development Gene 133

Outsourcing Pharma

AUGUST 31, 2021

According to patient-matching specialist TrialJectory, 90% of cancer patients know the vitality of genetic testing but more than half are not being tested.

Genetics 69

Genetics Clinical Development Development 69

Pharmaceutical Technology

OCTOBER 25, 2022

There are options in the future to possibly apply the worldwide research and development (R&D), manufacturing and marketing expertise of Astellas in gene therapy to AAV gene therapy development programmes of Taysha for genetic ailments of the central nervous system (CNS).

Gene Therapy 264

Gene Therapy Gene Development Genetics 264

Pharmaceutical Technology

MAY 17, 2023

The company will also be entitled to receive potential development and regulatory milestone payments, along with royalty payments. It will also handle the IND-enabling toxicology studies while Chinook Therapeutics will undertake the clinical development and commercialisation activities of the therapy.

Development 246

Development Protein RNA Genetics 246

Camargo

JULY 27, 2021

When developing a clinical development program for these increasingly popular therapies, it is important that sponsors use modeling and pharmacokinetic (PK) analysis to evaluate parameters that can be measured while dosing with gene therapy drugs, to characterize exposure-response data and inform rational dosing.

Gene Therapy 238

Gene Therapy Gene Gene Editing Genetics 238

pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. Alternatively, a drug can be administered that switches off the genetic medicine and brings protein expression to a halt. Vertex is also offering up to $1.3

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Pharmaceutical Technology

MAY 17, 2023

Takeda rare genetics and haematology therapeutic area unit head Daniel Curran said: “There is a critical need for treatment options for people living with cTTP, an ultra-rare, life-threatening disorder that has no therapies specifically approved for prophylactic treatment.

Genetics 242

Genetics Clinical Development Trials Drugs 242

Outsourcing Pharma

JULY 23, 2020

A leader from the genetic services company discusses how use of telemedicine can help pull in patients and keep trials going.

Genetics 52

Genetics Trials Clinical Development Development 52

Drug Discovery Today

JULY 28, 2020

Cancer Research UK, the University of Southampton and Touchlight Genetics, a London based biotechnology company, today (Wednesday) announce a new clinical development partnership to progress a therapeutic DNA vaccine, TGL-100, into an early phase clinical trial targeting head and neck squamous cell carcinoma (HNSCC).

Genetics 40

Genetics Vaccination Vaccine Development 40

The Pharma Data

NOVEMBER 30, 2020

(Nasdaq: NVAX), a late-stage biotechnology company developing next-generation vaccines for serious infectious diseases, today provided an update on its COVID-19 vaccine program. About NVX-CoV2373 NVX-CoV2373 is a protein-based vaccine candidate engineered from the genetic sequence of SARS-CoV-2, the virus that causes COVID-19 disease.

Clinical Development 52

Clinical Development Vaccination Vaccine Development 52

Pharmaceutical Technology

MAY 24, 2023

Streamlining the regulatory process, the AMP BGTC will also facilitate cost-efficient vector production to increase access for patients with rare and ultra-rare genetic diseases. The collaboration creates a reusable standardised method that minimises upfront costs and helps to reduce development barriers.

Gene Therapy 242

Gene Therapy Gene Development Manufacturing 242

Pharmaceutical Technology

FEBRUARY 22, 2023

Biohaven Clinical Development vice-president Lindsey Lee Lair said: “We are very pleased the FDA granted Fast Track designation for taldefgrobep alfa for the treatment of SMA. According to Biohaven, SMA affects nearly one in 11,000 births in the US, and it is a genetic carrier in approximately one in every 50 Americans.

Clinical Trials 246

Clinical Trials Clinical Trials Genetics Protein 246

Pharma Mirror

OCTOBER 26, 2021

Leveraging the clinical development expertise of cellular and genetic technology developed by Castle Creek, Mayo Clinic principal investigator, David R. The partnership was forged under the leadership of Castle Creek President and CEO, Mathew Gantz, and direction from Founder and Chairman Jeff Aronin. Deyle, M.D.,

Gene Therapy 130

Gene Therapy Gene Genetics Clinical Development 130

Pharma Marketing Network

DECEMBER 21, 2020

Almost two decades after the human genome was sequenced, a trickle of new genetic medicines (i.e., those that modify the expression of an individual’s genes or repair abnormal genes) has entered clinical practice, including 11 RNA therapeutics, 2 in vivo gene therapies, and 2 gene-modified cell therapies.

Genetics 52

Genetics Medicine Gene Therapy Marketing 52

Pharmaceutical Technology

MAY 17, 2023

Boundless Bio intends to use the funds for the initial clinical development of the first ecDNA-directed therapy (ecDTx), BBI-355, through clinical data readouts from its ongoing Phase I/II POTENTIATE clinical trial. The company is developing the assay with SOPHiA GENETICS.

Clinical Trials 147

Clinical Trials Clinical Trials DNA Genetics 147

Pharmaceutical Technology

MAY 19, 2023

ImmunoForge will expedite the development of new drugs based on the elastin-like polypeptide (ELP) platform technology originally developed by Duke’s Professor Ashutosh Chilkoti. ImmunoForge chief technology officer Dr Jim Balance pioneered the clinical development of peptide drugs genetically fused to ELPs.

Development 130

Development Drugs Licensing Licensing 130

Outsourcing Pharma

JANUARY 18, 2024

Inceptua Group and Cycle Pharmaceuticals partnership will give access to Free Goods Partnership that gets medicine to patients with extremely rare genetic conditions, which they would be unable to access otherwise.

Genetics 95

Genetics Medicine Clinical Development Development 95

Drug Discovery World

JULY 18, 2023

A study into long Covid has provided the first detailed genetic insights into the condition and its commonalities with other diseases, including myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS). The analysis identified 73 genes that were highly associated with severe and fatigue dominant forms of the disease.

Genetics 52

Genetics Drugs Gene Clinical Development 52

Pharmaceutical Technology

AUGUST 24, 2022

Prevail will also handle the complete production, clinical development and marketing activities. Lacerta is entitled to receive an upfront payment as well as development and commercial milestone payments from Prevail. In January last year, Lilly acquired biotechnology company Prevail for nearly $1.04bn.

Development 130

Development Gene Therapy Genetics Gene 130

Scienmag

JUNE 8, 2021

Data supports QurAlis’ approach to treat hyperexcitability induced neurodegeneration in ALS patients; QurAlis gears up for clinical development of therapeutic candidate, QRL-101 CAMBRIDGE, Mass.–(BUSINESS

Genetics 75

Genetics Clinical Development Medicine Development 75

Pharmaceutical Technology

MARCH 2, 2023

QN-023a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for Relapsed Acute Myeloid Leukemia. QN-023a overview QN-023a is under development for the treatment of relapsed or refractory acute myeloid leukemia (AML).

Gene Editing 100

Gene Editing Genetic Engineering Engineer Clinical Trials 100

Pharmaceutical Technology

DECEMBER 15, 2022

These targets will be selected with genetic validation and mechanistic hypotheses, preclinical validation and subject stratification biomarkers to streamline the assets’ clinical development. Additionally, the drug discovery capabilities of PrecisionLife will be used to offer a clear rationale to Ono for choosing targets. .

Genetics 130

Genetics Development Clinical Development Drugs 130

Pharmaceutical Technology

MARCH 2, 2023

QN-019a is under clinical development by Hangzhou Qihan Biotechnology and currently in Phase I for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia). QN-019a overview QN-019a is under development for the treatment of relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL).

Gene Editing 100

Gene Editing Clinical Trials Clinical Trials Genetics 100

The Pharma Data

OCTOBER 21, 2020

and Shionogi Limited as shareholders, today announced the positive findings of a pooled analysis of COVID-19-related impacts across the investigational long-acting cabotegravir and rilpivirine clinical development programme. This step is essential in the HIV replication cycle and is also responsible for establishing chronic infection.

Clinical Development 52

Clinical Development Development Medicine Clinical Trials 52

Pharmaceutical Technology

DECEMBER 14, 2022

GSK has entered a strategic partnership with Wave Life Sciences to progress the discovery and development of oligonucleotide therapies for new genetic targets. The alliance will merge the PRISM oligonucleotide platform of Wave and the capabilities of GSK in genetics and genomics.

Life Science 162

Life Science Development Gene Therapy Genetics 162

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. It is certainly a pivotal moment for the cell and gene industry, with the next decade of R&D and clinical trials expected to transform the future of medicine.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

Pharmaceutical Technology

MARCH 1, 2023

These therapies validated antisense technology as a new platform for drug discovery and for targeting rare genetic disorders. RNA-based therapeutics are a rapidly growing category of drugs that may improve the standard of care (SOC) for many diseases and could soon become the mainstay of personalised medicine.

RNA 147

RNA Vaccination Vaccine Genetics 147

Drug Discovery World

APRIL 24, 2024

Evotec will identify best-in-class small molecules targeting a key fibrotic pathway with strong genetic support identified by Variant Bio and progress the programme towards the selection of a clinical development candidate(s) using its R&D platform.

Development 52

Development In-Vivo In-Vitro Genome 52

Roots Analysis

FEBRUARY 27, 2024

In addition, over 430 gene therapy candidates are presently in different stages of clinical development, for which over 500 clinical studies are currently underway in various regions across the globe.

Manufacturing 52

Manufacturing Gene Therapy Gene DNA 52

Pharmaceutical Technology

AUGUST 17, 2022

Merck will also make royalty payments on any approved products developed out of the partnership. The rights to the oRNA-LNP technology platform of Orna will be retained by the company, which will also progress various other fully owned programmes in oncology and genetic disease areas.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

The Pharma Data

DECEMBER 13, 2020

As part of the collaboration, Hummingbird will be leveraging Tempus’ AI-enabled platform and proprietary data, as well as joining its TIME Trial® Network, for rapid identification, site activation and efficient enrollment of cancer patients who have NRG1 fusions and meet eligibility criteria for HMBD-001 clinical trials.

Clinical Development 52

Clinical Development Medicine Development Antibody 52

Drug Discovery World

MAY 17, 2023

Leveraging historical trial databases has become commonplace in assessing the feasibility of trials in therapeutic areas with a large breadth of clinical development history. EHR data also has a key role to play in designing future clinical trials.

Clinical Trials 52

Clinical Trials Clinical Trials Medicine Trials 52

Drug Discovery World

MAY 17, 2023

Leveraging historical trial databases has become commonplace in assessing the feasibility of trials in therapeutic areas with a large breadth of clinical development history. EHR data also has a key role to play in designing future clinical trials.

Clinical Trials 52

Clinical Trials Clinical Trials Trials Genetics 52

Drug Discovery World

JULY 6, 2023

The drug candidate, a monoclonal antibody (mAb) targeting the protein Semaphorin-3A (Sema3A), is being developed as a potential first-to-market treatment for Alport syndrome, a rare genetic kidney disease. Semaphorin-3A is an extracellular guidance protein and a well-known regulator of the actin cytoskeleton.

Antibody 52

Antibody Trials Genetics Protein 52