Clinical Development and Genetics - Clinical Research Informer

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Worldwide Clinical Trials

JANUARY 24, 2024

Written By: Derek Ansel, MS, CCRA, Executive Director, Therapeutic Strategy Lead, Rare Disease Given that 80% of rare diseases have a genetic etiology, genetic implications should be addressed at the onset of a clinical program to support trial enrollment. One diagnostic example that I discussed in my presentation is autism.

Genetics

Genetics Trials Clinical Trials Clinical Trials

Hemab raises $135m in Series B cash to treat genetic bleeding disorders

BioPharma Reporter

FEBRUARY 21, 2023

The Danish-US biotech Hemab Therapeutics bags $135m to finance the clinical development of prophylactic treatments for genetic bleeding disorders including Glanzmann thrombasthenia and von Willebrand disease.

Genetics

Genetics Clinical Development Development

SOPHiA GENETICS and AstraZeneca partner for oncology drugs development

Pharmaceutical Technology

FEBRUARY 13, 2023

SOPHiA GENETICS is expanding its collaboration with AstraZeneca to include multimodal approaches for developing cancer drugs. This collaboration aims to use the global SOPHiA DDM platform, a cloud-native platform of SOPHiA GENETICS, and multimodal algorithmic capabilities for AstraZeneca’s oncology portfolio.

Genetics

Genetics Development Drugs Genome

Webinars

Bridging Innovation & Patient Care: The Growing Role of AI

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Alkeus raises funds to launch Stargardt disease therapy gildeuretinol

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

Genetics Life Science Trials Clinical Development

To Share or Not to Share Failed Genetic Screening Results with Patients

Worldwide Clinical Trials

FEBRUARY 6, 2023

When using genetic screening to identify clinical trial volunteers, a sponsor’s obligations for further testing and disclosure of results to patients and families are unclear, especially when the results have no impact on medical management. Guidance in this area is much needed. Read the full article!

Genetics

Genetics Clinical Trials Clinical Trials Genotype

Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

FDA Law Blog

NOVEMBER 10, 2024

The Clinical Trials Grants Program provides funding for clinical trials that evaluate the safety and efficacy of potential treatments for rare diseases to help move promising treatments through clinical development. This study design is both innovative and efficient, leveraging a single-arm, baseline-controlled design.

Production

Production Clinical Development Development Regulation

Interferon alfa-2b by Center for Genetic Engineering and Biotechnology for Genital Warts (Condylomata Acuminata): Likelihood of Approval

Pharmaceutical Technology

SEPTEMBER 8, 2024

Interferon alfa-2b is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase II for Genital Warts (Condylomata Acuminata).

Genetic Engineering

Genetic Engineering Engineer Genetics Clinical Development

Astellas to support development of Taysha’s gene therapy programmes

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

Gene Therapy Gene Development Genetics

ESCAPE Bio Snags $73 Million to Advance Neurodegenerative Clinical Development Programs

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

Clinical Development Development Genetics Medicine

LC-002 by Genetic Immunity for Human Immunodeficiency Virus (HIV) Infections (AIDS): Likelihood of Approval

Pharmaceutical Technology

DECEMBER 20, 2023

LC-002 is under clinical development by Genetic Immunity and currently in Phase II for Human Immunodeficiency Virus (HIV) Infections (AIDS).

Genetics

Genetics Clinical Development Development

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

Pharmaceutical Technology

AUGUST 18, 2022

The US Food and Drug Administration (FDA) has granted approval for bluebird bio ’s Zynteglo (betibeglogene autotemcel, beti-cel) for the treatment of the underlying genetic cause of beta?thalassemia thalassemia in adult and paediatric patients.

Gene Therapy

Gene Therapy Gene Genotype In-Vivo

CIGB-128 by Center for Genetic Engineering and Biotechnology for Brain Tumor: Likelihood of Approval

Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. It was also under development for the treatment of mycosis fungoides and recurrent or progressive malignant glioma. It is a combination of interferon alfa-2b with interferon gamma.

Genetic Engineering

Genetic Engineering Engineer Genetics Clinical Trials

CIGB-128 by Center for Genetic Engineering and Biotechnology for Brain Tumor: Likelihood of Approval

Pharmaceutical Technology

JUNE 10, 2023

CIGB-128 is under clinical development by Center for Genetic Engineering and Biotechnology and currently in Phase I for Brain Tumor. It was also under development for the treatment of mycosis fungoides and recurrent or progressive malignant glioma. It is a combination of interferon alfa-2b with interferon gamma.

Genetic Engineering

Genetic Engineering Engineer Genetics Clinical Trials

Chinook and Ionis partner to develop ASO therapy for kidney disease

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

Development Protein RNA Genetics

Precision eye therapy for dogs ready for human clinical development

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

Clinical Development Gene Therapy Development Gene

FDA grants priority review to Takeda’s BLA for cTTP therapy

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

Genetics Clinical Development Trials Drugs

FDA grants Fast Track status for Biohaven’s taldefgrobep alfa to treat SMA

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

Clinical Trials Clinical Trials Genetics Protein

Forge Biologics joins BGTC for new AAV gene therapies development

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

Gene Therapy Gene Development Manufacturing

Survey: cancer patients missing out on much-needed genetic testing

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

Genetics Clinical Development Development

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. 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

Gene Therapy Gene Medicine Genetics

Castle Creek Biosciences Collaborates with Mayo Clinic to Expand Gene Therapy Platform

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

Gene Therapy Gene Genetics Clinical Development

Boundless Bio raises funds to advance ecDNA-directed therapies

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

Clinical Trials Clinical Trials DNA Genetics

Lilly’s Lepodisiran Achieves Over 90% Reduction in Heart Disease Marker in Phase II Trial

XTalks

APRIL 3, 2025

Eli Lilly shared new Phase II trial data showing that its experimental siRNA therapy, lepodisiran, leads to reductions in levels of lipoprotein(a) (Lp(a)) a genetically inherited risk factor for heart disease in adults with elevated levels of the marker. High Lp(a) levels can double or even triple the risk of a heart attack.

Trials

Trials Clinical Trials Clinical Trials Genetics

InformedDNA: telemedicine connects genetic patients to clinical care

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

Genetics Trials Clinical Development Development

ImmunoForge expands agreement with Duke University for drug development

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

Development Drugs Licensing Licensing

Lacerta partners with Prevail to develop AAV capsids for CNS diseases

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

Development Gene Therapy Genetics Gene

GSK and Wave Life Sciences partner to develop oligonucleotide therapeutics

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

Life Science Development Gene Therapy Genetics

Frontotemporal dementia: the state of treatment development

Pharmaceutical Technology

FEBRUARY 20, 2023

Different approaches that are studied include antisense oligonucleotides (ASOs), and gene therapies, which are in early clinical trials. Other lines of research look at the genetic overlap between FTD and amyotrophic lateral sclerosis (ALS), which could be used in the development of treatments for both conditions.

Development

Development Gene Therapy Protein Gene

Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

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

Gene Editing Genetics Drugs Gene

Cell Reports publishes data supporting the importance of ion channel, Kv7.2/7.3 as a target in ALS

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

Genetics Clinical Development Medicine Development

Novavax Announces COVID-19 Vaccine Clinical Development Progress

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

Clinical Development Vaccination Vaccine Development

Merck and Orna partner for RNA technology-based vaccines and therapies

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

RNA Vaccination Vaccine In-Vivo

PrecisionLife and Ono sign multi-target partnership for CNS disorders

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

Genetics Development Clinical Development Drugs

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

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

Genetics Medicine Gene Therapy Marketing

Cancer Research UK, University of Southampton and Touchlight Genetics Partner to Develop Head and Neck Cancer Vaccine

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

Genetics Vaccination Vaccine Development

Psychiatry Clinical Trials: Inclusive, Patient-Centric Approaches for Mental Health Research

XTalks

DECEMBER 19, 2024

Finally, psychiatry clinical trials must also address the variability in treatment responses. Each patients experience with mental health conditions is influenced by unique genetic, psychological and environmental factors.

Clinical Trials

Clinical Trials Clinical Trials Trials Research

Inceptua and Cycle partnership brings hope to patients at the end of the treatment road

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

Genetics Medicine Clinical Development Development

Enhancing Access and Retention in Global, Cross-Border Rare Disease Clinical Trials

XTalks

APRIL 8, 2025

There are over 10,000 rare diseases affecting an estimated 300 million people worldwide where 80% are genetic , 95% lack approved treatments and nearly half begin in childhood. Cross-border enrollment captures a wider range of genetic backgrounds, environmental influences, and cultural perspectives.

Clinical Trials

Clinical Trials Clinical Trials Trials Gene Therapy

Patient Safety and Radiopharmaceuticals Administration: Minimizing Radiation Exposure and Risks

Worldwide Clinical Trials

JULY 8, 2024

With radiopharmaceuticals emerging as a diagnostic and therapeutic (theranostics) procedure, many are in the clinical development pipeline and are expected to play a crucial role in the future of healthcare. Genetic Factors : Certain genetic mutations, such as those in DNA repair genes, can affect how cells respond to radiation.

Hormones

Hormones Regulation Clinical Trials Clinical Trials

QN-023a by Hangzhou Qihan Biotechnology for Relapsed Acute Myeloid Leukemia: Likelihood of Approval

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

Gene Editing Genetic Engineering Engineer Clinical Trials

6 Principles of Our Rare Disease Methodology That Drive Success for Our Sponsors

Worldwide Clinical Trials

MAY 14, 2024

As the strategists for Worldwide Clinical Trials’ rare disease sponsors, we design customized solutions that make a difference in achieving successful outcomes. Our approach is informed by our combined 30+ years in rare disease clinical development and our time in various stakeholder roles, such as scientists, CRAs, and project managers.

Trials

Trials Clinical Trials Clinical Trials Research

QN-019a by Hangzhou Qihan Biotechnology for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia): Likelihood of Approval

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

Gene Editing Clinical Trials Clinical Trials Genetics

Astellas teams up with UK biotech Mogrify on hearing loss project

pharmaphorum

JULY 5, 2022

It can be caused by exposure to loud noises – including prolonged use of earbuds at high volumes – as well as cancer treatment, illnesses, genetic mutations, or aging. SNHL is the most common form of hearing loss, account for more than 90% of cases and affecting millions of people worldwide. An estimated 1.57

In-Vivo

In-Vivo Bioinformatics Genetics Licensing

How can we fulfill the promise of Cell and Gene Therapy?

Pharma Mirror

JANUARY 30, 2022

Some 3,366 treatments are currently in preclinical and clinical development[1]. He explains how we can navigate obstacles to ensure C&GT can achieve its full potential. As a treatment area, C&GT has shown robust growth in recent years. The global market is projected to reach $13.8 billion by 2026 from $7.7

Gene Therapy

Gene Therapy Gene Clinical Development Marketing

Leveraging Genetic Testing for Enrolling Rare Disease Trials

Hemab raises $135m in Series B cash to treat genetic bleeding disorders

Webinars

Trending Sources

SOPHiA GENETICS and AstraZeneca partner for oncology drugs development

Webinars

Alkeus raises funds to launch Stargardt disease therapy gildeuretinol

To Share or Not to Share Failed Genetic Screening Results with Patients

Regulator and Funder? FDA’s Orphan Products Grants Program awards significant funding to help move promising treatments through clinical development

Interferon alfa-2b by Center for Genetic Engineering and Biotechnology for Genital Warts (Condylomata Acuminata): Likelihood of Approval

Astellas to support development of Taysha’s gene therapy programmes

ESCAPE Bio Snags $73 Million to Advance Neurodegenerative Clinical Development Programs

LC-002 by Genetic Immunity for Human Immunodeficiency Virus (HIV) Infections (AIDS): Likelihood of Approval

FDA grants approval for bluebird’s Zynteglo to treat beta-thalassemia

CIGB-128 by Center for Genetic Engineering and Biotechnology for Brain Tumor: Likelihood of Approval

CIGB-128 by Center for Genetic Engineering and Biotechnology for Brain Tumor: Likelihood of Approval

Chinook and Ionis partner to develop ASO therapy for kidney disease

Precision eye therapy for dogs ready for human clinical development

FDA grants priority review to Takeda’s BLA for cTTP therapy

FDA grants Fast Track status for Biohaven’s taldefgrobep alfa to treat SMA

Forge Biologics joins BGTC for new AAV gene therapies development

Survey: cancer patients missing out on much-needed genetic testing

Cell and gene therapies: why advanced medicines call for specialised logistics

Castle Creek Biosciences Collaborates with Mayo Clinic to Expand Gene Therapy Platform

Boundless Bio raises funds to advance ecDNA-directed therapies

Lilly’s Lepodisiran Achieves Over 90% Reduction in Heart Disease Marker in Phase II Trial

InformedDNA: telemedicine connects genetic patients to clinical care

ImmunoForge expands agreement with Duke University for drug development

Lacerta partners with Prevail to develop AAV capsids for CNS diseases

GSK and Wave Life Sciences partner to develop oligonucleotide therapeutics

Frontotemporal dementia: the state of treatment development

Vertex eyes controllable genetic drugs with $1.3bn Obsidian alliance

Cell Reports publishes data supporting the importance of ion channel, Kv7.2/7.3 as a target in ALS

Novavax Announces COVID-19 Vaccine Clinical Development Progress

Merck and Orna partner for RNA technology-based vaccines and therapies

PrecisionLife and Ono sign multi-target partnership for CNS disorders

Advances in Genetic Medicine May Be Outpacing Some Clinicians’ Understanding, But Pharmaceutical Marketers Can Do Much to Address the Problem

Cancer Research UK, University of Southampton and Touchlight Genetics Partner to Develop Head and Neck Cancer Vaccine

Psychiatry Clinical Trials: Inclusive, Patient-Centric Approaches for Mental Health Research

Inceptua and Cycle partnership brings hope to patients at the end of the treatment road

Enhancing Access and Retention in Global, Cross-Border Rare Disease Clinical Trials

Patient Safety and Radiopharmaceuticals Administration: Minimizing Radiation Exposure and Risks

QN-023a by Hangzhou Qihan Biotechnology for Relapsed Acute Myeloid Leukemia: Likelihood of Approval

6 Principles of Our Rare Disease Methodology That Drive Success for Our Sponsors

QN-019a by Hangzhou Qihan Biotechnology for B-Cell Acute Lymphocytic Leukemia (B-Cell Acute Lymphoblastic Leukaemia): Likelihood of Approval

Astellas teams up with UK biotech Mogrify on hearing loss project

How can we fulfill the promise of Cell and Gene Therapy?

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