QLT announces clinical & regulatory update for oral retinoid program for inherited retinal disease

QLT announced that, following meetings with the U.S. Food and Drug Administration and the European Medicines Agency, the Company believes that it is close to finalizing a pivotal trial protocol for QLT091001 for the treatment of inherited retinal disease such as Leber Congenital Amaurosis (LCA) and Retinitis Pigmentosa (RP) due to mutations in the LRAT and RPE65 genes, both orphan indications.

The Company expects to provide final guidance on its development plans in these indications before the end of the first quarter of 2014 after final feedback from the European regulatory agency.

Additionally, QLT has initiated recruitment of subjects for a Phase IIa proof-of-concept trial of its drug candidate, QLT091001, in adult subjects with Impaired Dark Adaptation (IDA), a condition that results in decreased ability to recover visual sensitivity in the dark after exposure to bright lights.

The Company also announced the launch of a compassionate use program for QLT091001 in LCA and RP, as well as plans for a patient registry and an update on its retreatment study in these indications.

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Govt accepts Ranjit Roy panel report on approval of new drugs, clinical trials & banning of drugs

The Union Health Ministry has accepted the recommendations of the Prof. Ranjit Roy Chaudhury expert committee.  The committee was constituted by the ministry in February this year to formulate policy and guidelines for approval of new drugs, clinical trials and banning of drugs.

According to senior officials in the ministry, the recommendations of the expert committee were discussed in a meeting with its members recently. During the meeting, clarifications on certain recommendations were obtained from the committee. After the meeting, the ministry in-principle accepted the recommendations of the committee. 

Accepting the recommendations of the expert panel for accreditation of ethics committees, investigators and the clinical trial sites, the ministry decided that in order to strengthen the clinical evaluation of new drugs, the clinical trials should be conducted in accredited sites by accredited investigators with the oversight of accredited ethics committees (Ecs). As this is a long term measure, in the meantime, Quality Council of India (QCI) will be considered for creating a system for accreditation of investigators, ethics committee and clinical trial sites. Although the Drugs & Cosmetics Rules, 1945, already provide for registration of ethics committee, accreditation of such committees will be undertaken following a specific procedure. 

As this requires amendments in the Drugs & Cosmetics Rules, as an immediate measure, CDSCO would initiate steps relating to the process of accreditation by constituting an expert body of 20-25 experts. The names of experts will be finalized by CDSCO in consultation with Dr. Ranjit Roy Chaudhury, Dr. Y. K. Gupta, Prof. & Head, Dept. of Pharmacology, AIIMS, New Delhi and Dr. Arun Aggarwal, Prof. of ENT, Maulana Azad Medical College, New Delhi, senior officials in the ministry said. 

Accepting the recommendations of the committee on the procedure for review of applications of clinical trials and new drugs, officials said that the New Drug Advisory Committees (NDACs) will be renamed as Subject Expert Committees. The members for their meetings will be drawn randomly from a large pool of experts. Applications of clinical trials and new drugs will initially be evaluated by the Subject Expert Committees and their recommendations will be reviewed by the Technical Review Committee (TRC). The TRC will be constituted under DGHS and consisting of experts from each areas i.e. clinical pharmacology, regulatory clinical toxicology/ pathology, medicinal/ pharmaceutical chemistry, pharmacy and immunology including clinicians, basic scientists involved in drug development and subjects specialists (drug indication wise). CDSCO will grant approval of clinical trial and new drugs based on the recommendations of TRC.

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UT Arlington researchers exploring better methods for gene therapies to fight the sight-deteriorating disease Retinitis Pigmentosa.

UT Arlington researchers are exploring a better method for initiating certain gene therapies that could better fight the sight-deteriorating disease retinitis pigmentosa. This research is funded by the National Institutes of Health.

Dr Samarendra Mohanty, Assistant Professor of Physics, at UT Arlington, is focused on using near-infrared ultrafast laser beam to deliver genes that allow expression of light-sensitive proteins, called opsins, into specific cells. That proteins’ expression allows researchers to influence neural activity through optical or light stimulation – a technique known as optogenetics.

In the past, the genes have been delivered to cells by virus. That method can have drawbacks, such as immune responses, in addition to the benefits. In Mohanty’s method, a laser beam creates a transient sub-micrometer size hole, which allows for the gene encoding the proteins to permeate through the cell membrane. It can limit the risk of immune response, as well as delivering larger genes than viral methods, he said.

The scientists claim that with the minimally invasive near-infrared method, DNA and other impermeable molecules can be effectively delivered only to the places most required. In retinitis pigmentosa, the peripheral retina begins to lose light sensitivity due to loss of photoreceptors. With this method, a laser can deliver the genes only to the required part of the retina, making those neurons respond to light again. This method will be more effective than a virus, where the genes are delivered everywhere, potentially causing complications in areas that are already working fine.

Optogenetic stimulation also holds promise for influencing neurons in the brain. Scientists, including Mohanty’s research group, are researching ways it could be used to understand how the brain works or to intervene in case of neurological disorders or to affect behavior.

Ultimately, Mohanty’s team has a goal of creating all optical, or light-based, control and monitoring of cell activity. So, in addition to the light-assisted delivery of genes, the researchers also will work on refining methods for stimulating the neural activity using near-infrared and visible light. Some of those methods are described in a recently published paper called “Fiber-optic two-photon optogenetic stimulation,” which appeared in the journal Optics Letters.

Mohanty’s lab at UT Arlington also will use a method called phase-sensitive interferometry, to monitor the changes in neurons that result from the activation by light. The interferometry method is called “label-free” because unlike fluorescence, it uses the change in behavior of light rays, rather than staining, to track changes at the sub-nanometer level.

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